Issuu

p.13 | DIABETES ALERT

p.17 | HELP FOR AGING JOINTS

p.24 | WASHINGTON DAYS 2025

Risk rises with age. Early screening and prompt treatment are key.

For older men with hemophilia, exercise and proactive care are essential.

The annual event highlights the power of collective advocacy.

The Bleeding Disorders Magazine

| hemaware.org

PROPOSED FEDERAL CUTS COULD DISRUPT ACCESS TO LIFESAVING CARE FOR PEOPLE WITH BLEEDING DISORDERS

Sounding the Alarm p.18

SPRING / SUMMER 2025

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Sponsored Content

Harnessing Community Insights to Shape the Future of Hemophilia B Research At Regeneron, we believe it’s essential to truly understand the communities we aim to serve. By listening to the voices of those affected by diseases— patients, caregivers, and healthcare providers—we ensure our research focuses on their unique challenges as we strive to address real unmet needs.

Laying the Groundwork: Disease Research as the Cornerstone of Development In hemophilia B, effectively addressing a challenge first requires a deep understanding of underlying disease biology. The standard of care currently requires frequent, ongoing medical treatment, which can be difficult on patients and families. This led our focus on the genetic drivers of the condition. Shifting research paradigms starts with rigorous preclinical research. Meagan O’Brien, Senior Medical Director, explains how “leaving no stone unturned during the preclinical phase means building solid foundations of scientific evidence through detailed testing—part of our responsibility to build confidence and trust within the community.”

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Rege Hem Sponsored Content

Developing a Shared Lexicon “Helping standardize discussions around gene editing can equip patients with the same, accurate information to promote informed consent,” said Michaela Jones, Director and Medical Affairs Lead for the hemophilia B program.

The lexicon creates consistent, inclusive, and understandable language for discussing gene editing among patients and care partners— demystifying complex scientific concepts with detailed descriptions and visual aids.

Infusing Patients’ Perspectives into the Clinic

Patient panels organized by the Regeneron Patient Advocacy team provided crucial input on clinical trial design, educational materials, and even resulted in specific protocol adjustments to be better tailored to the needs of the hemophilia B community. As one example, feedback resulted in removing trial participant age limits to promote broader enrollment of a representative population.

According to Virginie Delwart, Director of Patient Advocacy, “Patient feedback shaped clinical protocols to not only embody scientific rigor but reflect the community’s needs and expectations to better direct our research toward the right challenges, instilling a sense of partnership.”

Fostering Community-Centric Collaboration Prioritizing early, continuous engagement with the community via advocacy groups helped integrate patient voices into key decisionmaking processes, placing their experiences and concerns at the forefront of clinical initiatives. Relationships with organizations like the National Bleeding Disorders Foundation and the European Hemophilia Consortium remain crucial in gathering

feedback to evolve our programs to the needs of the community—through events like family education days, national conferences, and more. Regeneron is conducting ongoing research in hemophilia B. Study drugs have not been reviewed by any regulatory authority and their safety and effectiveness are unknown. Talk to your doctor to learn more about your treatment options.

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With Regeneron investigating novel approaches like CRISPR gene editing, we sought to provide clarity where uncertainty may arise—partnering with several advocacy groups and leading medical voices to develop a comprehensive lexicon.

Engaging with patient groups early–with insights gathered over a year and half prior to entering the clinic–illuminated a clearer picture of the realities the community faces, which were then infused into our clinical development programs.

2 pag

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Bindin Sadd Inks:

Inside |

H E M AWA R E M A G A Z I N E

Living Well 13

Mind & Body

Could you be at risk for diabetes? Early screening and prompt treatment are key.

Family Matters

Find out what you can do to help make your child’s playdates successful and safe.

Generation Next

Here’s why you should consider disclosing your bleeding disorder to your inner circle of friends.

Women’s Health

Learn why women with bleeding disorders may find themselves at an increased risk for stroke.

In Our Prime

For older men, exercise and proactive care are essential to managing joint pain.

In Every Issue 5

EXECUTIVE LETTER

Our collective voice is powerful. 7

HEALTHY START

How to avoid summer safety hazards, all about patient assistance programs, and what to know about 504 Plans.

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C OV E R : I L LU S T R AT I O N BY A N G E L A KO C H .

6/13/25 12:39 PM

SPRING/SUMMER 2025

Cover Story 18 Sounding the Alarm

Proposed federal cuts could disrupt access to lifesaving care for people with bleeding disorders. Learn how our community is responding — and what you can do to help ensure a future of health, equity, and hope.

Community in Action 24 NBDF Update

27 Global Focus

30 P ipeline

26 Chapter

28 Philanthropy

31 My Journey

More than 400 volunteer advocates from 49 states participated in Washington Days 2025.

Roundup

Chapters are leading state efforts to ban copay accumulator adjuster policies.

More people around the world now have access to diagnosis, treatment, and care.

Matters

Generous contributions are helping to fund research into better treatments and improved care.

Learn about three new subcutaneous treatment options for hemophilia A and B. The Igielinski brothers aren’t letting hemophilia keep them from pursuing their dreams.

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My Journey to Jivi® invites you to learn about the inspiring, relatable stories of our speakers as they share the defining moments that shaped their lives.

Austin Caldwell

Venkat Reddy

Brandon Schrom

Juan Pablo Lopez Padilla

Ryan Moulton

Zach Castillo

Our speakers have been compensated by Bayer to share their stories.

Scan the QR Code to Learn More © 2025 Bayer. All rights reserved. Bayer, the Bayer Cross, and Jivi are registered trademarks of Bayer. 05/2025 PP-JIV-US-2301-1 Bayer, 100 Bayer Blvd, Whippany, NJ 07981 1-888-84-BAYER (1-888-842-2937)

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hemaware.org

President and CEO Philip M. Gattone, M.Ed.

In Focus PRESIDENT AND CEO PHIL GATTONE, M.ED.

Chair of the Board Ryan Griffith

Vice Chair of the Board Susan Hartmann

Treasurer Joseph Alioto, M.D.

ISSUE CONTRIBUTORS

Proposed legislation and other government changes threaten to disrupt access to care for millions of Americans.

Cheryl Ashmore; Christina Bond; Samantha Carlson; Deniece Chevannes; Melissa Compton; Javey Dallas, MSW, LCWS; Annette von Drygalski, M.D.; Nancy Durben; Gabi Flores; Sharice Flores; Lauren Holomalia; Alex Igielinski; Max Igielinski; William Lankford; Stephanie Lapidow; Jennifer Newman; Emily Ouellette; Rich Pezzillo; Wendy Price; Doris Quon, M.D.; Sarah Richardson; Bill Robie; Dawn Rotellini; Nathan Schaefer; Kathaleen Schnur, MSW, LCSW; Bridget Tyrey; Michael Viscariello, LCSW; Peter Zdziarski

Executive Editor Kyla Clark

Advertising Sales Tom Corcoran, thomasWcorcoran@proton.me, 215.275.6420

Contributing Staff Associate Angelina Wang

MANIFEST.COM

EDITORIAL, DESIGN & PRODUCTION MANAGEMENT Managing Editor Donna Behen, donna.behen@manifest.com

Associate Creative Director Tami Rodgers

Senior Editor Matt Morgan

Art Director Angela Koch

Production Director

P H OTO G R A P H Y BY N ATA L I A W E E DY.

Laura Marlowe HemAware (ISSN 1088-7008) is published two times annually by the National Bleeding Disorders Foundation (NBDF), 1230 Avenue of the A mericas, 16th Floor, New York, NY 10020, 212.328.3700, toll free 800.42.HANDI, fax 212.328.3777. The opinions expressed are those of the authors alone and do not represent the opinions, policies, or position of NBDF, the Board of Directors, or editorial staff. This material is provided for your general information only. NBDF does not give medical advice or engage in the practice of medicine. NBDF under no circumstances recommends particular treatments for specific individuals and in all cases recommends that you consult your physician or local treatment center before pursuing any course of treatment. All manuscript submissions should be typed double space, between 500 and 1,000 words in length and sent by email to donna.behen@manifest.com. HemAware retains the right to edit submissions. The content of the article is the responsibility of the author(s) and must be original work that has not been, nor will be, published elsewhere. A copyright agreement attesting to this and transferring copyright to NBDF is required. Acceptance of advertising for products and services in HemAware in no way constitutes endorsement by the National Bleeding Disorders © Foundation. 2025 National Bleeding Disorders Foundation.

Our Collective Voice Is Powerful The bleeding disorders community has a long history of using their collective voice to speak out against issues that negatively impact patients and families. One of the best examples is the passage of the landmark R icky Ray Hemophilia Relief Fund Act in 1998, which provided compensation to people (and their families) who had acquired HIV from contaminated clotting factor products. Integral to the passage of the bill was the involvement of a united and dedicated group of people with hemophilia and their family members who came to Washington, D.C., to meet with their elected off icials and tell their stories to help move the legislation forward.

In this issue’s cover stor y on page 18, you’ll read about proposed legislation and other government changes that threaten to disrupt access to care for millions of Americans — including thousands in the bleeding disorders community. Changes to Medicaid — the nation’s largest health insurer for low-income individuals — will impact 30% of people with bleeding disorders in the U.S., leaving them more vulnerable to developing long-term complications such as joint damage and disability. This is not a time to remain on the sidelines. It’s time for all of us to come together with strength and unity and carry forward the advocacy that represents the essence of who we are as a community.

Online | hemaware.org National Bleeding Disorders Foundation: bleeding.org Facebook: @NationalBleedingDisordersFoundation X: @nbd_foundation and @NBDFespanol Instagram: @nbd_foundation TikTok: @natlbdfoundation

YouTube: youtube.com/@NBDFvideo Advocacy: bleeding.org/advocacy Steps for Living: stepsforliving.bleeding.org Victory For Women: victoryforwomen.org Better You Know: betteryouknow.org

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Body language can tell you all sorts of things. Like someone is having a stroke.

SPEECH DIFFICULTY

FACE DROOPING

ARM WEAKNESS

strokeassociation.org

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TIME TO CALL 911

Know the sudden signs.

Spot a stroke F.A.S.T.

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6 9:31 AM

Healthy Start S TAT S , FAC T S , A N D N E W S YO U C A N U S E

The Power of Words We asked community members, “What’s a quote or mantra that helps you stay motivated on tough days?” Here are some of your answers:

Small, shaky steps still count.

G E M M A C A N F LY / S TO C K S Y.

—CHERYL ASHMORE

It will not define us and our lives. —SARAH RICHARDSON

Rare diseases aren’t given to those who deserve them, they are given to those who can handle them.

You can do hard things. —SHARICE FLORES

—PETER ZDZIARSKI

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Healthy Start |

S TAT S , F A C T S , A N D N E W S Y O U C A N U S E

The out-of-pocket costs of

a program. Whenever possible,

medications for people with

ask your current insurer to

bleeding disorders can be

cover your factor, seek a new

exorbitant. Finding out that you

insurance company, or use

can’t afford your factor because

Medicaid or Medicare coverage,

of health insurance changes

if applicable.

may make you feel hopeless or

— Lisa Fields

helpless. But patient assistance programs (PAPs) may help you manage your bleeding disorder

scan here

until you find another way to get

NBDF’s Neil Frick Resource Center has information about manufacturers’ patient assistance programs, with details about medications, costs, eligibility, and enrollment.

your essential medication. Many drug manufacturers

K KG A S , LU M I N A / S TO C K S Y.

WHAT YOU SHOULD KNOW ABOUT PATIENT ASSISTANCE PROGRAMS

offer PAPs for a limited time, as a stopgap measure. They provide pricey medications at no cost or significantly reduced rates. Some people seek PAPs when they switch jobs and have a gap in health insurance coverage. Other people need help because their insurer stopped covering their factor. Still others don’t earn enough to pay their health insurance premiums or purchase prescriptions. PAPs aren’t long-term solutions, and there can be gaps in care while you wait to be accepted to

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BDC 2025 BY THE NUMBERS

NBDF’s 77th Bleeding Disorders Conference will be held Aug. 21-23, 2025, at Gaylord Rockies Resort & Convention Center in Aurora, Colorado. Here’s a brief look at what you can expect.

2,000+ 120+ 100+ 35+ attendees

ASK A SOCIAL WORKER

What is a 504 Plan, and does my child need one?

A 504 Plan is a formal educational plan designed to provide students with disabilities the support and accommodations they need to succeed academically. Legally, all public schools must provide students with disabilities a 504 Plan upon request. For a child with a bleeding disorder, a 504 Plan can outline accommodations such as permission to take breaks when needed, avoiding activities that increase the risk of injury, modifying physical education requirements to avoid strenuous activities, and access to medical care during school hours. Additionally, teachers and staff would

be informed of the child’s needs and trained to respond appropriately in the event of a medical emergency. While the severity of a child’s bleeding disorder may vary, a 504 Plan is highly recommended to ensure your child’s health and well-being are supported so that they can thrive academically. — Michael Viscariello, LCSW Viscariello is a social worker at the Hemostasis and Thrombosis Center at Children’s Hospital Los Angeles, and a member of the Social Work Working Group.

scan here Learn more about the laws that exist to help your child get the best education possible.

sessions and presentations

speakers with 5 sessions for children 9 to 12 years old

exhibitors

scan here Learn more and register.

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Connect with an ® ALTUVIIIO Peer Mentor ALTUVIIIO Peer Mentors are real patients or caregivers who have had similar experiences to yours.

Maybe it’s because I’ve always had such great support, but engaging with others is the best way for me to stay happy.

You’ll have the opportunity to: • Ask questions • Hear firsthand experiences • Learn helpful tips • Get to know others in the community

—AJ AJ | ALTUVIIIO patient AJ is a promotional speaker compensated by Sanofi.

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Healthy Start |

S TAT S , F A C T S , A N D N E W S Y O U C A N U S E

The Top Summer Safety Hazards and How to Avoid Them Nothing takes the fun out of your summer like being sidelined by a heat-related condition that could have been prevented. SPF 30+

When you plan a trip to the beach or the mountains, you pack factor for yourself or your child, a first-aid kit, and other essential supplies. Taking additional precautions should help keep you safe from common summer pitfalls such as:

D E H Y D R AT I O N

FOOD POISONING

SUNBURN

Being active during hot weather makes you sweat, which may cause dehydration.

Leaving your packed lunch or picnic spread out for hours may make you sick.

Every time you get a sunburn, it increases your lifetime risk of skin cancer.

S O L U T I O N Replenish your fluids often to stay hydrated. Take water with you whenever you go out, and sip regularly. Make sure that your kids do, too — they may become too distracted to remember to drink. If you wait to drink until you feel thirsty, you may already be getting dehydrated.

S O L U T I O N Keep cold items on ice to reduce the risk of bacteria contaminating your food. Refrigerate uneaten food sitting at room temperature within two hours. If your buffet is outside in the heat, chill your leftovers as soon as possible, and dispose of anything that seems spoiled.

S O L U T I O N To decrease that risk, slather yourself and your child with sunscreen. Choose a product that’s water-resistant, with broad-spectrum protection, and an SPF level of 30 or higher. Reapply every two hours that you’re outside. For additional protection, avoid the sun during peak hours (10 a.m. to 4 p.m.), spend time in the shade, and wear a wide-brimmed hat.

— Lisa Fields

CHILL LEFTOVERS

G I L L I A N VA N N / S TO C K S Y.

H Y D R AT E

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KEEP CALM AND AND

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LIVING WELL

M I N D & B O DY

Symptoms of Diabetes Frequent urination Excessive thirst Extreme fatigue Blurred vision Frequent headaches Cuts or sores that are slow to heal

SILENT DISEASE Many people don’t recognize that they’re having symptoms until diabetes becomes more serious.

Could You Be at Risk for Diabetes?

C H E L S E A V I C TO R I A / S TO C K S Y.

To prevent complications, early screening and prompt treatment are key When Christine Bond was diagnosed with hemophilia B in her late 20s, she began to prioritize her health, starting with losing weight. Yet as she got older, dropping pounds seemed impossible. “I lost 15 pounds but gained it back super-fast,” Bond, 47, recalls. “I just felt so old. And tired.” Then an endocrinologist diagnosed her with insulin resistance (also known as prediabetes). For Dawn Rotellini, chief operating officer of the National Bleeding Disorders Foundation, a diabetes diagnosis in 2014 on top of her hemophilia came as a shock. “I went in for my annual physical and they ran my labs. The doctor said, ‘Your A1C is concerning, but we think this is a mistake,’” says Rotellini, 61. A repeat test confirmed she had prediabetes. Within two years, it progressed to diabetes.

RISK INCREASES WITH AGE Hematologist Doris Quon, M.D., medical director of the Hemophilia Treatment Center at the Luskin Orthopaedic Institute for Children in Los Angeles, says improved treatments help people with hemophilia live longer, but that means they have to pay attention to the diseases that come with aging — including diabetes. Many aren’t even aware that they are at risk, or even having symptoms, until it becomes more serious. “Patients mention that they have to urinate all the time, and that they’re always thirsty,” she says, noting the classic symptoms. “It’s very important to get treated because it affects the kidneys so you could end up needing dialysis, and you may also develop neuropathy [a loss of feeling in your hands and feet], or wound healing and vision problems.” “The thought of losing a foot or my vision from diabetes is a scary thing. I’m trying to avoid it,” says Bond, a tax preparer from Edgewood, Maryland. She initially went on Metformin, a diabetes drug, but eventually switched to Ozempic, which helped her lose weight.

Rotellini found O zempic made her nauseous. She switched to Trulicity, and her numbers have improved. She also got a glucose monitor, which “absolutely changed everything for me” because it helped her track her glucose levels throughout the day. BE SCREENED Many people with bleeding disorders rely on their hematologist for all of their health care, but Quon urges them to see a primary care physician for screening, especially if they are overweight or have a family history of diabetes. If you do have diabetes, lifestyle changes — such as avoiding processed and sugary foods (especially soda), eating more vegetables, and exercising — are as important as medication. The side benefit, Quon says, is your joints will be healthier. Bond saw that firsthand. “Now that I weigh less, my target joints aren’t as problematic.” —By Andrea Atkins

scan here Learn more about diabetes and other common health concerns that occur as you age.

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LIVING WELL

FA M I LY M AT T E R S

Preparing for Playdates Here’s how to nurture those social connections while also balancing safety concerns

TALK HONESTLY WITH PARENTS

Tell them about your child’s bleeding disorder, letting them know that it’s controlled with medication and by avoiding high-risk activities. “Sometimes when people hear ‘bleeding disorder,’ they ’re overwhelmed,” says Kathaleen Schnur, MSW, LCSW, a social worker at the Hemophilia Center of Western Pennsylvania in Pittsburgh. “People with bleeding disorders don’t bleed any faster, they just bleed longer. Sometimes that normalizes it for people.” Give parents concrete examples of acceptable playdate activities. “Maybe your child can climb on things but only to a certain height, or they can shoot hoops, but you don’t want them using the monkey bars,” Ramirez says. For initial get-togethers, ask parents to stay, so you get to know each other.

FUN TIME Playdates help foster confidence and selfesteem.

“Consider what keeps your kid safe, but allow them the opportunity to socialize.” — K ATHALEEN SCHNUR, MSW, LCSW

For drop-off playdates, leave your phone number, telling parents to call or text with any concerns. PREPARE YOUR CHILD

Let your child know that they can ask the other parent for help or have them call you if there’s a problem. “Many kids know when a joint bleed is coming on, and if they start feeling that tingling or warm sensation, they should tell the parent that they need to call their mom or dad,” Ramirez says.

I wasn’t able to get there,” says Lauren Holomalia of Wai’anae, Hawaii, whose 13-year-old son Mason has severe hemophilia A. “Now that my son is older, he can self-infuse.” Encourage playdates early and often. “Consider what keeps your kid safe and gives you comfort,” Schnur says, “ but allow them the opportunity to socialize, listen to other grown-ups, and sort things out with their peers.” —By Lisa Fields

PLAN ACCORDINGLY

What you send with your child depends on the playdate’s length, the distance from home, and the severity of your child’s condition. Many parents send a first-aid kit, a nosebleed kit, or factor in a cooler. “I had a binder with easy, step-by-step directions, should anything happen and

scan here Read NBDF’s helpful guide for parents of children with a bleeding disorder on how to find childcare.

I L LU S T R AT I O N BY M I G U E L DAV I L L A .

When your child gets invited on playdates, they may be elated; you might feel nervous. Any parent may worry about another adult being in charge for a few hours. Parents of children with bleeding disorders have additional concerns, not knowing how someone else will handle a medical problem should it occur. “With a bleeding disorder, your first instinct is to do everything you can to keep your child safe,” says Lucy Ramirez, MSW, LCSW, a clinical social worker at Rush Hemophilia and Thrombophilia Center in Chicago, but playdates are an important part of childhood. “These activities foster self-esteem and confidence through fun experiences that help create emotional bonds,” Ramirez says. Use these strategies to encourage successful get-togethers:

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LIVING WELL

G E N E RAT I O N N E X T

What Friends Need to Know About Your Bleeding Disorder

WHO DO YOU LET IN?

Here’s why you should consider disclosing to your inner circle

FEELING SEEN Gabi Flores (at right) says talking about her bleeding disorder benefits her mental health.

Not everyone has to know. But if you were not able to advocate for yourself, who would you trust to step up for you? Look for the people who would act responsibly and in your best interests with the information. Ask yourself: Who are the people you tell your secrets to? Who do you trust to have your back if others are trying to get you to do things that you know aren’t safe for you? “Those are the people who see you as a whole person,” Dallas adds. WHAT INFORMATION SHOULD YOU GIVE?

Flores tells her friends about symptoms to watch out for. For example, when she was playing college soccer, she was on a medication that made her limit her water inta ke. If she drank too much, she would get symptoms that mimicked dehydration. She told her teammates about this, and they all watched out for her. She a lso ex pla ins things to her friends that they don’t have to worry about. “I’m an athlete, but my friends sometimes wonder if I should be playing that hard. I tell them I know my limits and what not to worry about.” —By Beth Levine

I M AG E C O U R T E S Y O F G A B I F LO R E S .

Friendship Is A Two-Way Street “I was diagnosed with a platelet disorder when I was 14,” says Gabi Flores, 24, of St. Louis. “I was very shy, but I had to tell my friends something because I was out of school a bit for doctor appointments. I’m glad I did because they all had my back and helped me.” When you have a bleeding disorder, the questions soon arise: Should you tell your friends? How much do you tell them? Whom do you trust with this very personal information? This issue becomes more critical as you reach young adulthood and become more independent from your parents.

WHY TELL YOUR TRUSTED FRIENDS?

Disclosing your bleeding disorder to others can be a crucial part of self-advocacy, says Javey Dallas, MSW, LCSW, a social worker at the Oklahoma Center for Bleeding and Clotting Disorders in Oklahoma City. “While your parents and loved ones watched out for you for all your life and were your advocates, now you need to start advocating for yourself so you can transition into independence,” he says. Telling others also has mental health benefits. “It does mean a lot to share that part of myself. It makes me feel seen and understood,” Flores says.

As the saying goes, everyone you meet is fighting a battle you know nothing about. Just as you seek normalcy and acceptance, so do your friends. Ask if you can help them with anything. “I try to put myself in other people’s shoes and consider that maybe I don’t see the full picture of what somebody is going through,

and there might be underlying factors,” says Gabi Flores. Social worker Javey Dallas urges you to create a culture of acceptance. “This will lend itself to forming the community and the friendships that you want. Being able to share allows you to shape not only your present, but also your future.”

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LIVING WELL

WO M E N ’ S H E A LT H

“Discuss the pros and cons with your doctor.” — ANNET TE VON DRYGALSKI, M.D.

What to Know About Stroke Prevention Women with bleeding disorders may be at higher risk Stroke is the No. 3 cause of death in women, according to the American Stroke Association (ASA). It’s a statistic that Wendy Price knows all too well. The 70-year-old Carlsbad, California, resident, who was diagnosed with von Willebrand disease at age 45, has multiple risk factors for stroke, including high blood pressure and a family history of cardiovascular disease. But updated stroke prevention guidelines released by the ASA in 2024 highlighted several additional risk factors for stroke that are unique to women, including pregnancy, taking oral cont racept iv es, or bein g on horm on e replacement therapy. The guidelines are particularly relevant to women with bleeding disorders since many of them take oral contraceptives to control heavy bleeding, says Annette von Drygalski,

M.D., Pharm.D., director of the Center for Bleeding and Clotting Disorders at the University of California San Diego. “In addition, hemorrhagic strokes, which occur when a blood vessel in or around the brain ruptures and causes bleeding into the brain, can be more severe in women with bleeding disorders,” von Drygalski says. HOW TO PROTECT YOURSELF

Here’s what women, girls, and people with the potential to menstruate (WGPPM) can do to lower their risk of stroke: Talk to Your Doctor About Estrogen. Oral contraceptives that contain estrogen (also known as combined hormonal contraception) have been shown to increase the risk of stroke in WGPPM even though the amount of estrogen is lower now than in the past. The risk is even greater if you also smoke. Estrogen replacement during

—By Beth Levine

scan here Learn more about the risk factors for stroke from the American Stroke Association.

J AV I E R PA R D I N A / S TO C K S Y.

ESTROGEN ALERT Both the Pill and hormone replacement therapy can increase the risk of stroke.

and after menopause can also heighten stroke risks, von Drygalski says. “If you take oral contraceptives to control heavy bleeding or you’re on hormone replacement for menopause, discuss the pros and cons with your doctor, especially if you have other risks such as advanced age, hypertension, or migraines,” she says. Other contraception options to consider include progestin-only birth control and intrauterine devices (IUDs). For relief of menopausal symptoms, ask your physician about transdermal patch applications of estrogen. “The risk is decreased if the medication is given through the skin, rather than a pill,” von Drygalski notes. Be Proactive. You c a n help reduce t he level of you r st roke r i sk on you r own. Exercise moderately in a safe manner for the sever it y of your d isorder; practice good sleep hygiene; follow the Mediterranean diet; and monitor your cholesterol and blood pressure. If you have risk factors, von Drygalski recommends getting evaluated by a cardiologist, even at a young age. Cardiologists should be encouraged to discuss patient-specific stroke risks and prevention strategies with the patient’s hematologist. To protect herself, Wendy Price now takes a cholesterol-lowering statin plus a blood pressure medication and monitors her levels regularly. She also exercises daily to help keep both those numbers down.

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IN OUR PRIME

LIVING WELL

How Men Can Manage Joint Pain as They Age Exercise and proactive care can keep pain in check As men w ith hemophilia get older, many grapple w ith increasing joint pain — a serious consequence of repeated bleeds. Half of men age 50 and older cited “worsening of joint disease and joint pain” as one of their greatest future concerns in a National Bleeding Disorders Foundation (NBDF) survey. Jennifer New man, a physical therapist at the U NC Hemophilia and Thrombosis Center in Chapel Hill, North Carolina, says improving chronic pain treatment for people with bleeding disorders is a top priority. “So much contributes to a person with a bleeding disorder’s experience with chronic pain, ranging from changes in the weather, a poor night’s sleep, insurance issues, new shoes, stress, and so much more,” Newman says. “It’s a lot more than only joint damage.”

Strength Training

Balance Mangement

PA I M AG E S A R C H I V E / C O N T R I B U TO R / G E T T Y I M AG E S .

Aquatic Therapy

ADVANCING YEARS TAKE A TOLL Older men with bleeding disorders are especially susceptible to joint pain. Newman says recurrent bleeding episodes cause progressive damage to the bone, cartilage, and muscle. Combine this with agerelated osteoarthritis, and diagnosis and treatment can become trickier. “For someone with a history of joint bleeds, these age-related changes can hit harder, leading to muscle weakness, stiffness, and changes in movement that impact daily life,” she explains. This most commonly affects the ankles, knees, and elbows. That’s certainly the case for William Lankford, a 59-year-old with hemophilia A. “You learn to live with the pain,” he says. “But you also learn what actually works for you with trial and error over time.”

PAIN MANAGEMENT STRATEGIES Newman says there’s no one-size-fits-all approach, but certain evidence-based options can help reduce pain. “A program focusing on fall prevention and balance, strength training, and main-

taining functional mobility is a great place to start,” she says. Fear of movement is common, says Nancy Durben, a physical therapist at the Hemostasis and Thrombosis Center at Oregon Health & Science University, but she reminds patients that they can strengthen their muscles at any age. “Getting older does mean we may need to pay attention to our bodies differently and make adjustments to keep ourselves healthy and strong,” Durben says. The key is combining exercise, like aquatic therapy, with weight management, medication, and psychosocial support. Older patients can especially benefit from physical therapy treatments such as elastic taping and bracing. Lankford says he relies on movement, nonsteroidal anti-inflammatory drugs, and regular visits with his physical therapists to help him reduce pain and increase mobility. His secret weapon: He doesn’t travel anywhere without a hair dryer. “It can take a pain that’s almost unbearable away. It’s just one of the many tricks I’ve learned to manage without serious painkillers,” he says. —By Celeste Sepessy

“Getting older does mean we may need to pay attention to our bodies differently.” — NANCY DURBEN

DIVE IN Swimming is a great way to strengthen muscles.

scan here The Steps for Living website has a wealth of information about aging well with a bleeding disorder.

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ADVOCACY ALERT

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ADVOCACY ALERT

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ADVOCACY ALERT

On May 22, 2025, the U.S. House of Representatives narrowly

prioritizes national goals in tax policy, defense, and immigration, and pr As a result, key health programs — including Medicaid and other vital p

A preliminary analysis by the independent Congressional Budget Office (CBO) projects that the Medicaid provisions in the bill could lead to

10 million people losing coverage by 2034.

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narrowly passed H.R. 1, the “One Big Beautiful Bill Act.” The legislation , and proposes substantial budget reductions to fund those investments. r vital public health services — face significant restructuring. Change is a constant in public policy. But for millions of Americans living with complex and chronic health conditions, the nature and speed of that change matter deeply. What’s at stake is not only fiscal balance, but continuity of care, access to treatment, and — ultimately — quality of life. At the National Bleeding Disorders Foundation (NBDF), we understand the immense responsibility of crafting sustainable, future-focused health policy. We also understand the human cost when vulnerable populations lose access to the support systems they depend on. Together with our partners, we are working to raise awareness and promote constructive dialogue to protect the health and dignity of those we serve. MEDICAID IN THE CROSSHAIRS

One of the most consequential aspects of the bill is its proposed restr ucturing of Medicaid — the nation’s largest health insurer for low-income individuals. A mong the proposed changes are new work reporting requirements for certain adult enrollees, revisions to eligibility and enrollment processes, and reduced federal matching funds for the 40 states that expanded Medicaid access. The bill also introduces cost-sharing measures such as copays for services and prescriptions, which could create additional barriers to care. The se pol ic y sh i f t s a re not merely a d m i n i s t rat ive. According to an early analysis by the Congressional Budget Office (CBO), they could result in up to 10 million people losing coverage by 2034. The bleeding disorders community is particularly at risk. Approximately 30% of individuals with bleeding disorders are covered by Medicaid, relying on high-cost biologic treatments — sometimes multiple times per week — to prevent dangerous internal bleeding. Without access to this care, long-term complications such as joint damage and disability become inevitable. In a May 2025 letter to Congress, NBDF and allied organizations emphasized that changes to Medicaid must not come at the expense of human health and safety. “Introducing administrative complexity into an already fragile system risks pushing vulnerable patients out of care,” the letter warned.

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The expiration of Affordable Care Act subsidies will cause marketplace premiums to increase by an average of 75%, triggering

coverage losses for over 4 million Americans. These concerns are also shared by our partners in the sickle cell community, who face parallel challenges in treatment access and care continuity.

The bill also outlines a set of changes to the A ffordable Care Act (ACA), including adjustments to subsidy eligibility and the discontinuation of enhanced premium tax credits after 2025. Without these enhanced credits — introduced during the pandemic to increase affordability — marketplace premiums are expected to rise by an average of 75%, potentially leading to over 4 million Americans losing coverage. Other proposed changes, such as shortened enrollment windows, may further complicate access. For families managing rare and chronic conditions, including bleeding disorders, the ACA’s marketplace often fills critical coverage gaps. Limiting access to this safety net could mean fewer people getting the care they need — when they need it most. The bleeding disorders community has a history of success in this area. “In 2017, NBDF (then NHF) was instrumental in securing bipartisan support opposing the various ACA repeal and replace measures,” says Nathan Schaefer, senior vice president of public policy and access at NBDF. FAC E S B E H I N D T H E P O L I CY

Behind every policy debate are individuals and families whose lives will be directly affected. A child with hemophilia who receives weekly infusions to prevent painful bleeds. A young adult with von Willebrand disease who has transitioned off a parent’s insurance. A person living with factor X deficiency, navigating crisis episodes

that require specialized care. These are the human stories at the core of our advocacy. In a letter signed by several bleeding disorders advocacy groups, national leaders noted: “The proposed Medicaid reforms risk limiting access to outpatient care and essential treatments. For many, this could mean more hospitalizations, greater disability, or worse.” The same letter raised concern over the bill’s impact on the new CMS Cell and Gene Therapy Access Model, which seeks to make groundbreaking treatments more accessible through Medicaid. Without stable coverage pathways, participation in such forwardthinking programs becomes uncertain. FEDERAL AGENCIES IN TRANSITION

Alongside the proposed legislation, recent restructuring at the Department of Health and Human Ser vices (HHS) has caused

S E R G I O M E N D O Z A H O C H M A N ; B E A S T 0 1 ; C H R O N A O L E N A : F I LO ; A N D R Y DJ U M A N TA R A ; T R A F F I C _ A N A LY Z E R / G E T T Y I M AG E S .

E V O LV I N G T H E A F F O R D A B L E C A R E A C T

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I N PA R A L L E L W I T H L E G I S L AT I V E R E F O R M , R E S T R U C T U R I N G AT T H E D E PA R T M E N T O F H E A LT H A N D H U M A N S E R V I C E S ( H H S ) H A S S E N T S H O C K WAV E S T H R O U G H T H E P U B L I C H E A LT H S Y S T E M .

340B REFORM AND ACCESS T O M E D I C AT I O N S

The bill also includes reforms to the 340B Drug Pricing Program, which enables safety-net providers to offer discounted medications to patients who need them most. While many of these reforms aim to improve transparency and reduce misuse, some could unintentionally limit the ability of small hospitals and clinics to provide affordable care. Proposals to report profit margins and reduce certain reimbursements raise important questions about long-term sustainability. NBDF supports accountability in public health spending — but we also believe that reforms must be carefully calibrated to avoid disrupting care for underserved populations. T H E PAT H F O R WA R D : C O L L A B O R AT I O N ,

uncer tainty for federal public health programs. In April 2025, NBDF and more than 90 national and regional partners submitted a letter to HHS Secretary Robert F. Kennedy Jr. raising concerns about recent changes at the Centers for Disease Control and Prevention (CDC) and the Health Resources and Services Administration (HR SA) that may af fect bleeding disorder sur veillance and maternal-child health initiatives. Programs supported by the CDC’s Division of Blood Disorders and HRSA’s Hemophilia Program have long played a vital role in advancing public health through research, education, and safety monitoring. While modernization is important, continuity is critical. As the April letter stated, “Efforts to streamline government must preserve the services that improve care and save lives.”

N O T C O L L AT E R A L D A M A G E

As this issue goes to press in early June 2025, the One Big Beautiful Bill Act is under review by the U.S. Senate. NBDF remains committed to advancing solutions that support both fiscal responsibility and patient well-being. We know that policies must evolve. But the path forward must not leave our most vulnerable behind. “With unity and perseverance, we can prevent these drastic cuts and ensure the health and well-being of all members of the bleeding disorders community,” Schaefer says. We invite policymakers, agencies, and fellow advocates to work with us. The bleeding disorders community has always embraced innovation and partnership — across political lines and in service to shared values of equity and access. This is a moment to lead with hope, not fear. With compassion, not conf lict. And with a shared resolve to ensure that no one is left without the care they need to live a full and healthy life.

what you can do Scan the QR code to learn how you can join NBDF’s efforts to protect health coverage, support agency programs, and ensure that reforms work with patients, not against them.

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N B D F U P DAT E

Washington Days 2025 Bleeding disorders advocates in action on Capitol Hill

North Dakota advocates and NBDF President and CEO Phil Gattone with Matthew Lee (center), a staffer for Rep. Julie Fedorchak.

I M AG E S C O U R T E S Y O F N B D F C H A P T E R S .

On March 6, more than 400 dedicated advocates representing the bleeding disorders community gathered in Washington, D.C., for the National Bleeding Disorders Foundation’s (NBDF) annual Washington Days. Participants engaged in 248 Congressional meetings — nearly evenly divided between Democratic and Republican offices — covering 49 states and Puerto Rico. This year’s event highlighted the power of collective advocacy, as individuals and families shared compelling personal stories and called on lawmakers to protect access to comprehensive care, affordability, and essential health programs. These face-to-face conversations served not only to educate policymakers but also to strengthen the foundation of bipartisan support for the community’s most pressing policy priorities. Washington Days is more than a policy event — it’s a powerful reminder of the strength and unity within our community. From first-time advocates to seasoned policy champions, every conversation helped put a human face on the critical issues facing those living with bleeding disorders. With Capitol Hill as a backdrop, Washington Days served as a powerful reminder: Real change starts with real voices. These efforts help shape a future where everyone — regardless of diagnosis — can access the care they need.

scan here Sign up for NBDF’s newsletter for the latest updates on advocacy priorities, research, treatments, and educational events.

Nebraska Sen. Deb Fischer with Makenna Dietrich and her daughter Illa.

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Washington Days is more than a policy event — it’s a powerful reminder of the strength and unity within our community.

Guam Rep. James Moylan (second from left) with NBDF’s Dawn Rotellini, Bill Robie, and Nathan Schaefer.

Above, advocates from the Eastern and Western Pennsylvania Bleeding Disorders Foundations. At left, from left to right, Pennsylvania’s Keyai Lee, Melanie Rosen, Victoria Selinsky, and Charles Gilbert.

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CHAPTER ROUNDUP

The Power of State Advocacy Copay accumulator adjuster and maximizer policies pose a serious threat to people with chronic diseases by exposing them to high, unexpected costs and interrupting treatment. Advocating on the state level is critical to push through legislation to ban these policies.

ALL SMILES The Pederson family with North Dakota Gov. Kelly Armstrong.

“State advocacy is every bit as important as federal advocacy because states often have more authority over issues that impact people’s daily lives,” says Bill Robie, senior director of state government relations at the National Bleeding Disorders Foundation. Here are three chapters that are leading state efforts to ban copay accumulator adjuster policies.

HEMOPHILIA ASSOCIATION OF NEW JERSEY

GATEWAY HEMOPHILIA ASSOCIATION

The New Jersey Chapter regularly meets with legislators from across the state to educate them on what copay accumulator adjuster programs are and why they’re harmful to the bleeding disorders community. For Executive Director Stephanie Lapidow (below), sharing one’s own story about living with a chronic illness and the challenge of paying for necessary medications is key to getting the point across. “Advocacy is only as good as the personal stories shared with changemakers,” Lapidow says. “Anyone can speak passionately about an issue, but unless you have someone speaking up who has experienced it firsthand, it’ll only get you so far. Use your voice for good, and the change will follow.” Learn more about the

The Gateway Hemophilia Association (GHA) is currently supporting four pieces of legislation in Missouri to ensure all copays count toward a patient’s deductible. By participating in Washington Days, Missouri Advocacy Day, and collaborating with Bleeding Disorders Alliance Illinois on the first joint Illinois Advocacy Day, the chapter has made a strong impact on raising awareness and pushing for change. Bridget Tyrey, executive director of GHA, stresses that while participating in advocacy days is meaningful, a sustained commitment is what leads to lasting change. “Advocacy isn’t a day, but a continuous effort,” Tyrey says. “It’s about showing up consistently, listening, learning, and supporting those who need a voice.” Learn more about the Gateway

Hemophilia Association of New

gatewayhemophilia.org

Jersey: hanj.org

Hemophilia Association:

—By Christina Frank

Emily Ouellette, executive director of the Bleeding Disorders Alliance of North Dakota, has a husband with severe hemophilia, and their two daughters are carriers. “Without medication to aid in clotting, a minor injury for most people could be life-threatening to him,” Ouellette says. “North Dakota prides itself on being a business-friendly state, but failing to ban copay accumulator programs does not support businesses or employees,” she says. “It forces people out of the workforce due to untreated health conditions. When patients lose access to medication, they are at greater risk for medical complications, emergency hospital visits, and long-term disability, all of which drive up health care costs for families and employers alike. No patient should have to choose between affording their medication and providing for their family.” Learn more about the Bleeding Disorders Alliance of North Dakota: bdand.org

COURTESY OF NBDF CHAPTERS.

BLEEDING DISORDERS ALLIANCE OF NORTH DAKOTA

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G LO BA L FO C U S

PROGRESS Patients in India have better access to treatment and care.

Caring for the Global Community

C O U R T E S Y O F WO R L D F E D E R AT I O N O F H E M O P H I L I A .

Thanks to WFH USA, more people around the world now have access to diagnosis, treatment, and care World Federation of Hemophilia (WFH) USA plays a critical role in ensuring that people with inherited bleeding disorders receive the care and treatment they need — regardless of their type of bleeding disorder, gender, or where they live. By supporting the mission of the WFH within the United States, the organization engages American residents, foundations, and corporations to make a lasting impact through humanitarian aid; advocacy; training; education; research and data collection; and scholarships and grants.

WFH’s mission is being accomplished through several endeavors: The WFH Humanitarian Aid Program seeks to improve the lives of people with inherited bleeding disorders, giving them a quality of life that previous generations would not have thought possible. The program’s main support activities are delivering donated factor and non-factor replacement therapy, conducting training events, and providing logistics support. Since 1996, the program has helped more than 6,500 patients go on prophylactic treatment; distributed 2.3 billion

The global inherited bleeding disorders community is getting closer to the WFH vision of Treatment for All.

IUs of factor and 12.9 million milligrams of non-factor replacement therapy; and helped treat more than 30,000 people. The Women and Girls with Bleeding Disorders Initiative and the Global Policy and Access Summit provide the community with essential platforms to amplify voices and drive policy changes. These initiatives, along with several others, have reached over 130 countries. The WFH Cornerstone Initiative provides support, expertise, and training to countries where care for people with inherited bleeding disorders is severely limited or not available at all. A Cornerstone Initiative project is implemented for a period of up to four years, and then countries become eligible for the full range of programs and activities offered by the WFH. In 2 0 2 4 , t h e W F H i m p l e m e n t e d C o r n e r s t o n e In i t i a t i v e p r o j e c t s i n Madagascar, Rwanda, Tajikistan, Tanzania, and Togo. (Sierra Leone and Gabon will be joining in 2025.) Thanks to the initiative, these countries have been able to achieve significant progress in expanding diagnosis, outreach, and education. In Tanzania, there’s been a 2 1/2-fold increase in the diagnosed population since 2019. Training for health care professionals and improved governance structures in Rwanda have resulted in the identification of 26 new people with hemophilia. In Madagascar, the national member organization secured government approval to integrate hemophilia training into regional health care curricula, and in Tajikistan, the establishment of a hemostasis laboratory at the national reference hospital will provide free diagnostic testing to the community. In Togo, the WFH national member organization (NMO) organized training for board members, people with bleeding disorders, and health care professionals on outreach and identification strategies.

scan here Learn more about how WFH USA advances the global mission of the WFH in the U.S.

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P H I L A N T H R O PY

Chapter Support Fuels Fellowship Program Generous contributions help fund research into better treatments and improved care “Scientists in a lab don’t usually engage with community, but these researchers have come to our national conference,” Carlson says. “They’ve been able to interact with health care providers, patients, and families, which is why they’re in the business. They’re seeing their science impact real people, which is amazing and unique.” Since the JGP program began in 1972, NBDF has invested more than $9 million to fund 109 research fellows. In 2024, NBDF chapters donated $30,500 to JGP. Individual donors, sustainable gifts, and endowments from chapters provided additional support. “We simply couldn’t do this without the chapters, and we are incredibly grateful for their support” Carlson says.

NBDF aims to raise $125,000 for the JGP program in 2025.

HOPE IN ACTION JGP fellows have the freedom to study bleeding disorders in novel ways.

WHY CHAPTERS GIVE

Chapters support JGP for different reasons. “It’s part of our mission that we support research for better treatments and cures,” says Melissa Compton, executive director of the Lone Star Chapter. “Community members see the value in giving and continuing the research, especially those who have been around a while and have seen the treatments improve.”

A N I D I M I / S TO C K S Y. M I C R OVO N E / G E T T Y I M AG E S .

For more than 50 years, the National Bleeding Disorders Foundation (NBDF) chapters, family foundations, and families have funded the prestigious Judith Graham Pool (JGP) Postdoctoral Research Fellowship. The research program gives postdocs the freedom to study bleeding disorders in novel ways, so that they may develop better treatments and perhaps, one day, a cure. JGP fellows follow in the footsteps of the program’s namesake, Dr. Judith Graham Pool, who discovered in 1965 how to create cr yoprecipitate (concentrated clotting factor), forever changing the way that hemophilia was treated. “ W hen t he s e you ng s c ient i s t s get this award, they’re so excited,” says Samantha Carlson, director of research for the National Bleeding Disorders Foundation. “They become really dedicated to this community. The majorit y of them stay in the coagulation f ield. That is critical for recruitment and retention in our small community.” JGP fellows have opportunities to interact with people who have bleeding disorders, which is uncommon for researchers.

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“The JGP Fellowship, my first and only grant, was a great vote of confidence to a young researcher and served as the foundational basis and motivation for my continuing efforts in the biotech industry.” — GORDON VEHAR, PH.D., VICE PRESIDENT OF EX TERNAL INNOVATION, BIOMARIN PHARMACEUTICAL INC.

NBDF Chapters That Supported JGP in 2024/2025 PLATINUM: $25,000+ Hemophilia of Georgia

T h e Ne w E n g l a n d He m o p h i l i a Association (NEHA) informs their community about JGP fellows and their research efforts. “It’s important for families who are fundraising to know that part of their efforts are not only going to support NEHA’s vital programs and services, but they’re also going to support research,” says Rich Pezzillo, NEHA’s executive director. “The JGP program gives chapters like NEHA an opportunity to support future generations.” The Hemophilia of Georgia (HoG) Chapter hopes that its JGP contributions will make a meaningful difference in the world. “Some of us who work at HoG jokingly say, ‘We’re working to be out of work,’” says Deniece Chevannes, HoG’s vice president of public policy, health equity, and research. “We want to find a cure. Funding research is a way to deliver on that promise to patients.” —By Lisa Fields

scan here Make a donation to NBDF to support Judith Graham Pool Postdoctoral Research Fellowships.

VISIONARY: $10,000+ The Lone Star Chapter CHAMPION: $5,000+ Mary M. Gooley Hemophilia Center Greater Ohio Bleeding Disorders Foundation New England Hemophilia Association ADVOCATE: $1,000+ Bleeding Disorders Association of South Carolina Bleeding Disorders Foundation of North Carolina Gateway Hemophilia Association Hemophilia Association of the Capital Area Pacific Northwest Bleeding Disorders SUPPORTERS: UP TO $1,000 Kentucky Hemophilia Foundation Rocky Mountain Hemophilia & Bleeding Disorders Association Southwestern Ohio Hemophilia Foundation Virginia Hemophilia Foundation

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PIPELINE

New Approved Therapies for Hemophilia A and B These subcutaneous treatment options offer an alternative to traditional, intravenously infused factor replacement. designed to reduce or prevent the frequency of bleeding episodes. HYMPAVZI is the “f irst hemophilia medicine approved in the U.S. to be administered via a pre-filled, auto-injector pen,” according to Pfizer’s news release.

QFITLIA

ALHEMO

In Dec ember 2024, Novo Nord isk ’s Alhemo® (concizumab-mtci) was FDAapproved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 and older with hemophilia A or B with factor VIII or IX inhibitors. The daily subcutaneous therapy can be used to treat individuals with a hemophilia

MORE CHOICES Treatment options for adults with hemophilia have continued to expand.

In March 2025, the FDA approved Sanofi’s Qfitlia™ (fitusiran) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors. The subcutaneous therapy uses small interfering RNA (siRNA) technology to target antithrombin (AT), a liver-generated clotting protein that plays a key role in the regulation of blood clots. “Today’s approval of Q f itlia is significant for patients with hemophilia because it can be administered less frequently than other existing options,” said the FDA’s Tanya Wroblewski, M.D., in their March news release. “This new treatment option highlights our continued efforts to improve the lives of patients with hemophilia.” For more information about the potential side effects associated with the use of these products, please speak with your hematologist or primary care physician.

scan here Read the FDA news releases for these and other new products.

M A R C U S O N S A R I O / S TO C K S Y.

Pf izer’s HYMPAVZI™ (marstacimabhncq) received approval for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 12 and older with hemophilia A or B without factor VIII or IX inhibitors. “HYMPAVZI is a new type of drug that, rather than replacing a clotting factor, works by reducing the amount, and therefore, the activity of, the naturally occurring anticoagulation protein called tissue factor pathway inhibitor,” according to an FDA news release issued in October 2024. By increasing the amount of thrombin, an enzyme that is critical in blood clotting, the drug is

A inhibitor or a hemophilia B inhibitor. Like HYMPAVZI, it works by preventing tissue factor pathway inhibitor from performing the anticoagulant function that it naturally carries out in the human body. “The approval of Alhemo . . . provides a much-needed alternative to the current standard of care in hemophilia B with inhibitors, while offering patients with hemophilia A with inhibitors more treatment options, ultimately providing more patients with inhibitors the opportunity to personalize their care and address current treatment gaps,” Amy Shapiro, M.D., CEO and co-medical director of Innovative Hematology, said in Novo Nordisk’s news release.

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MY JOURNEY

NEW HEIGHTS Alex (left) and Max Igielinski with their dad, Chris, on top of Baldy Mountain.

The Sky’s the Limit

C O U R T E S Y O F T H E I G I E L I N S K I FA M I LY.

The Igielinski brothers don’t let hemophilia keep them from pursuing their dreams Alex: My twin brother and I each had a bleeding episode right after we were born. The doctors ran all sorts of tests and determined that we both had hemophilia. There’s no history of hemophilia anywhere in our family; it was due to a genetic mutation. Hemophilia probably impacted me the most at school — specif ically at recess, when kids play intense games like football, soccer, and gaga ball. I had to sit out most of the time because I didn’t want to get hurt. But that led

to oppor tunities for me to connect with other kids and broaden my friend group, which was really nice. Max: Alex and I have been in Scouts since we were 6 years old. We’re now 17. We can do everything other Scouts do, we just need to plan a little more, which is f itting for Scouts, since the mot t o is “Be Prepa red.” For exa mple, we went on a week long tr ip to Philmont Scout Ranch, a 214-squaremile stretch of rugged terrain, based in the Rocky Mountains in northeastern

“We can do everything other Scouts do, we just need to plan a little more, which is fitting for Scouts, since the motto is Be Prepared.” — MA X IGIELINSKI

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COMMUNITY IN ACTION

MY JOURNEY

New Mexico, where we climbed to the top of Mount Baldy. Alex: We planned for Philmont for two years. So much of the trip was about seeing how far we could go, but safely. We wanted to know that having hemophilia wouldn’t limit us. Max: Before we left, we had to think everything through. For example, we needed enough medicine, padded containers for the fragile vials, and special packs to keep the medicine at room temperature. In addition, we needed to figure out how we’d infuse ourselves safely in the middle of the wilderness. We used camping chairs to keep us off the dirty ground.

Alex: Our troop was prepared as well, since we’d sat down with them years ago. We’d explained our condition, and we described how they could help if necessary. For example, I said, “If you see me bang myself really hard, or it looks like I’m hurt in any way, tell my dad.” Ma x: It w a s re a l ly n ic e t o k now that I always had a huge community around me. And it’s helped to give me direction. One of the greatest things that Scouting America offers is merit badges. A big one for me was the First Aid badge, where I grew to love medicine. My merit badge counselor said I’d be a really good surgeon one day,

probably because I’ve been living the First Aid merit badge my entire life! Right now, my plan is to do some sort of pre-med major when I go to college. Al e x: Hemoph i l i a h a s pr e sent e d challenges, but it’s also brought me so much joy in different aspects of my life that I would have otherwise never been able to figure out. It was amazing to stand at the top of Baldy and take in the surroundings. I want people to know that hemophilia doesn’t have to rule their lives. I’ve gone beyond anything I ever thought I could do. —By Alex Igielinksi and Max Igielinski as told to Leslie Pepper

“Hemophilia has presented challenges, but it’s also brought me so much joy in different aspects of my life that I would have otherwise never been able to figure out.” — ALEX IGIELINSKI

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C O U R T E S Y O F T H E I G I E L I N S K I FA M I LY.

PROUD FAMILY Max (left) and Alex, shown with parents Chris and Amber and sister Aurelia, live in Cleveland.

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CELEBRATING ® 10 YEARS OF ALPROLIX SUPPORTED BY SANOFI’S COMMITMENT TO OUR COMMUNITY As it has for the past decade, our community continues to motivate and inspire us. That’s why we look forward to a future ﬁlled with possibility, backed by the community support that unites and raises us all.

CONNECT WITH A CoRe Sanofi Community Relations and Education (CoRe) Managers provide information about ALPROLIX, treatment options, and more. ©2024 Genzyme Corporation. All rights reserved. ALPROLIX and Sanofi are registered trademarks of Sanofi or an affiliate. MAT-US-2401992-v1.0-03/2024

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Hemophilia on your terms. Everyone’s hemophilia needs are different. We’re here to help meet those needs simply—with education and support options that can help inspire the new normal that’s just right for you.

Discover how we can help. RedefiningHemophilia.com

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