Reverse Rett Matters June 2023

June 2023

TRUSTEES

Chair: John H Sharpe Vice-Chair: Helen Simmonds

STAFF TEAM

CEO: Rachael Stevenson

Operations Manager: Catherine Coles Clinical Trial Lead/Supporter Care: Andy Stevenson

Design/Marketing: Wesley Havill

Patient Registry Co-ordinator: Ewelina Wasowska

Fundraising special events: Hazel Beiny Reverse Rett Matters Designer: Wesley Havill Edited by: Rachael Stevenson

Intro

Taysha news: World first for Rett Syndrome Gene Therapy

Neurogene Update

Reverse Rett Key numbers

Where does our regular income come from at Reverse Rett?

Does your child have a positive MECP2 mutation?

Call for expressions of interest

CIPP Centre Urgent Appeal

Jurassic Juggernauts

Reverse Rett Lakers

Meet our new Operations Manager

Sea to Summit

Do you live in or around London or the South East?

The Lives of Reilly

Why do we need to do so much fundraising at Reverse Rett?

Father's Day

Meet the Fire and Ice Maidens

The views expressed herein do not necessarily represent the views of the editor or of Reverse Rett Board of Trustees. Parts of this publication may be reproduced with permission, please email rachael@reverserett.org.uk

Dear Friends,

I hope you are enjoying the warmer weather and all the good things spring inevitably brings regardless of what else we have going on in our lives.

This is proving to be an interesting year for everyone who cares about Rett Syndrome research. The very first adult patient with Rett has now been dosed with Taysha’s gene therapy product in Canada. We are all holding our breath until we hear further news from Taysha Gene Therapies later this summer.

Neurogene are also now about to initiate a clinical trial, this time in children in the USA. You can read more about both trials and the full text of each company’s community letter in the following pages.

Despite all this promising news, there is still much work to do at Reverse Rett. We do not know how this trials will go, whether they will yield the much anticipated improvements in ability, function and health that we are all hoping for.

In the best case scenario we hope to see safety, and some efficacy at these early stages, which may enable regulators to allow companies to press on at speed.

In a different scenario, we may see no efficacy or a serious adverse event that stops a trial completely, returning companies and the scientists they’re working with back to the lab. The truth is that we simply don’t know until these experiments are done. Despite all the work that scientists and companies do ahead of human trials, some things will only be seen and learned from an actual human clinical trial.

That’s why we owe so much to the very first family who has taken this brave step We owe much as well to the families who will follow, to the young women who are in line to go next in the trial in Canada and all the US children who are up for screening.

I know first-hand, albeit from a drug trial rather than a gene therapy trial, that whilst taking part in a trial is a tremendous opportunity, it is a heart-wrenching experience as well. We want the people we love to be well and happy and seeing them enduring painful or invasive procedures and hospital appointments or admissions is difficult, especially when we are volunteering on their behalf.

But without their courage, without these families holding their nerve, the field cannot move on. We salute those patients, their families and carers, the scientists and companies working on the trials and you, our supporters and donors who have made it possible for us reach this critical moment in time.

All best wishes,

Rachael Stevenson, CEO, Reverse Rett

Rachael Stevenson, CEO, Reverse Rett

Taysha news: World first for Rett Syndrome Gene Therapy

The first person in the world to try gene therapy for Rett Syndrome has now been dosed with Taysha’s gene therapy product TSHA-102.

The news was announced by Taysha on 5th June 2023 in a press release and followed by a community letter which you can read overleaf.

This moment has been reached as a result of many years of work from scientists and researchers internationally.

Key take aways from the announcement

We do not yet know how the patient is faring after having had gene therapy but no news is good news from a safety point of view. We do not expect any further information about this until later in the summer.

Before any more patients can be dosed, the data from patient #1 must be reviewed by an independent data monitoring committee

This will be a minimum of six weeks after the first patient was dosed

The study is being conducted at CHU SainteJustine, the Université de Montréal mother and child university hospital centre in Montreal, Canada.

Plans to submit a UK Clinical Trial Application to the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK by mid-year are on track.

Details about the proposed age range for a UK trial are not available.

Plans to submit an Investigational New Drug Application to the UD Food and Drug Administration are on track for the second half of 2023 Again no details on age range.

Did you know?

The US based Rett Syndrome Research Trust supported by Reverse Rett, the Israeli Foundation for Rett Syndrome, Rett Syndrome Deutschland, OpéRett and more, funded the original academic work that led to the Taysha gene therapy program.

This funding which has mostly come from families and communities of people with Rett Syndrome, enabled the research to pass through the ‘valley of death’, which is the space between academic research and industry interest in developing the work into a commercial product.

Congratulations to Dr Steve Gray of UT Southwestern, now Chief Scientific Officer at Taysha Gene Therapies and to all the team at Taysha for all their ongoing work.

Taysha Community Letter

5th June 2023

Dear Rett Community,

Today, Taysha announced that the first patient was dosed with the investigational gene therapy, TSHA-102, in the REVEAL Adult Study, a clinical trial for females 18 years and older living with Rett syndrome.

We would like to thank the entire Rett community and the Rett patient advocacy groups for their partnership that supported this milestone. We would also like to acknowledge the family and individual who are participating in the trial for being the first to contribute to this important research in order to better understand the potential of gene therapy for Rett syndrome.

It is too early to make any conclusions about the potential safety or efficacy of TSHA-102 We will share additional information once data is collected and made public.

Please see below for a list of frequently asked questions.

What is the Reveal Adult Study?

The REVEAL Adult Study is a multi-center Phase 1/2 open-label dose-escalation clinical trial designed to evaluate the potential safety, tolerability, and preliminary efficacy of a single administration of the investigational gene therapy, TSHA-102, in adult females 18 years and older with Rett syndrome

The study is designed to evaluate two different dose levels to determine the optimal amount (highest dose without harmful side effects) of TSHA-102

During the dose finding part, study participants may receive one of two dose levels via a single intrathecal administration of TSHA-102 via lumbar puncture (spinal tap)

When will the second patient be dosed in the REVEAL Adult Study?

Taysha confers with an Independent Data Monitoring Committee (IDMC) to make decisions related to key clinical activities, such as proceeding with dosing a second participant.

The IDMC is an independent group of experts who monitor patient safety, potential efficacy and study conduct while a clinical trial is ongoing

IDMC members are selected based on their deep expertise, understanding of clinical trial methodology and ability to make sensible recommendations in a rational and consensusdriven manner based on data and medical judgment.

The IDMC will review initial data from the first participant before recommending to proceed with dosing a second participant; there will be an interval of at least six weeks.

Where is the REVEAL Adult Study clinical trial being conducted?

The study is being conducted at CHU SainteJustine, the Université de Montréal mother and child university hospital centre in Montreal, Canada.

Who do I contact for more information about the REVEAL Adult Study?

If you are interested in learning more about the study, please contact medinfo@tayshagtx.com

What are Taysha’s plans for a clinical trial for females with Rett syndrome in the United Kingdom (UK)?

Taysha plans to submit a Clinical Trial Application (CTA) to the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK by mid-year 2023 for the investigation of TSHA-102 in pediatric females with Rett syndrome; we do not yet have details on the specific age range.

Click here to read the Taysha Rett Community Letter from 1st February 2023

What are Taysha’s plans for a clinical trial for females with Rett syndrome in the United States (U.S.)?

Taysha plans to submit an Investigational New Drug (IND) application to the U.S. Food & Drug Administration (FDA) in the second half of 2023; we do not yet have details on the age range.

What is the underlying cause of Rett syndrome?

Rett syndrome is caused by a mutation in MECP2, which is a gene that makes a protein called MethylCpG-binding protein 2 (MeCP2). This protein plays an important part in the development of the brain throughout childhood by maintaining normal brain function and communicating between nerve cells.

In Rett patients with a MECP2 gene mutation, the body does not make enough MeCP2 or produces abnormal MeCP2.

What is Taysha’s investigational approach to gene therapy for Rett syndrome?

TSHA-102 is made up of a miniature form of the MECP2 gene (miniMECP2) that is paired with a regulatory element, or control, called miR The mini MECP2 gene is inserted into a delivery vehicle,or vector, which is then injected intrathecally, into the spinal fluid in the lower back (lumbar region).

The miRARE technology was developed by Dr Sarah Sinnet and Dr Steven Gray of UT Southwestern Medical Center and is designed to regulate or control the amount of MeCP2 protein that is made to avoid over expression.

Taysha’s clinical trials will determine whether the functioning mini MECP2 gene can be packaged with miRARE technology to safely regulate the level of MeCP2 protein expression.

If you have any additional questions or would like to connect with a member of our team, please contact medinfo@tayshagtx.com.

We are extremely grateful for the continued support of the Rett syndrome community and the Rett patient advocacy organizations. Your continued input helps to shape the work that we do to develop a treatment for Rett syndrome.

We look forward to providing further updates as new information becomes publicly available.

Sincerely,

The Taysha Patient Affairs Team

Neurogene publishes protocol for their children’s gene therapy trial due to start this year

In the last edition of this newsletter, we told you what milestones to look for over the summer.

One of them was Neurogene’s US clinical trial protocol for their product NGN-401 going up on the clinicaltrials gov website As of 12th June 2023, the protocol is up! Here are the key takeaways from the protocol:

The first confirmed enrolling trial site is Texas Children's Hospital

More sites are expected

Five female patients ages 4-10 in the first instance

Patients outside the USA cannot enrol at this time

Neurogene is working with regulators to add trial sites outside the USA

To read the full protocol please click here

Did you know?

Through our longstanding partnership with RSRT, Reverse Rett has provided a significant level of funding for Professor Stuart Cobb, now the Chief Scientific Officer of Neurogene, whose work led to the Neurogene NGN 401 program.

You can read Neurogene’s most recent letter to the community overleaf.

The Cobb Lab alone has received over £1.4million from Reverse Rett over the last decade enabling this work to move from the laboratory bench to human clinical trials.

We are delighted for Stuart and send our congratulations to him, all his colleagues at the Cobb Lab at the University of Edinburgh and all the team at Neurogene.

Neurogene Community Letter

June 12th 2023

Dear Rett Syndrome Community,

The purpose of this letter is to provide information about Neurogene’s Rett syndrome clinical trial and t communicate the first clinical trial site location.

This first-in-human investigational gene therapy clinical trial is titled: A Phase 1/2, Open-Label Clinical Study to Evaluate Safety, Tolerability, and Efficacy of NGN-401 in Pediatric Subjects with Rett Syndrome. This clinical trial will test NGN-401, an investigational adeno associated virus (AAV) gene therapy that contains a full-length human MECP2 gene and Neurogene’s Expression Attenuation via Construct Tuning (EXACT) transgene regulation technology, in females with Rett syndrome EXACT gene regulation is designed to express a controlled amount of MeCP2 protein.

The clinical trial is enrolling participants at Texas Children’s Hospital.

More details about the clinical trial and the site contact information are available at here

This link will be updated with clinical trial site information as additional site locations are opened in the future.

Since this is the first time the investigational gene therapy NGN-401 will be given to humans, the clinical trial will start with a small group of 5 participants. After safety assessments in this group are complete, we will make an informed decision about how to expand the clinical trial for additional participants to enroll.

About the Phase 1/2 Investigational Gene Therapy Clinical Trial for Rett Syndrome

This is a prospective, open-label clinical trial, which means all participants will receive the investigational gene therapy, NGN-401, and will be followed for 5 years, with an additional long-term follow-up for 10 years.

The investigational gene therapy, NGN401, will be given as a single intracerebroventricular (ICV) injection into a ventricle of the brain.

This clinical trial will study the investigational gene therapy, NGN-401, in 5 females aged 4-10 years old, with a diagnosis of typical Rett syndrome with a documented disease-causing mutation in the MECP2 gene

Participants are similar in age and stage of disease progression to better understand the safety and clinical effects of the investigational gene therapy.

Individuals with normal hand function will be excluded (e.g., holding a pen/pencil effectively and/or drawing a shape).

Individuals who are participating in another clinical trial for an investigational medicine will not be eligible for enrollment.

Each participant will be followed for safety and efficacy for 5 years after dosing.

For approximately the first 3 months after the investigational gene therapy is given, families will be required to live (or temporarily relocate) within a 2-hour drive of the clinical trial site.

Living near the clinical trial site is important to monitor safety and for multiple inperson follow-up visits required during the first three months.

After the initial safety monitoring period, there will be telephone and in-person visits with the clinical trial site in decreasing frequency over the rest of the 5-year period

After the 5-year period, it is expected that participants will enroll in a separate long-term observational study that will continue to collect information on safety and effects of the investigational treatment for 10 additional years.

Can families living outside of the United States enroll in the US clinical trial?

Not at this time We are early in the process of working with regulators on the opportunity to add clinical trial sites outside the US. We will provide further information once it becomes available.

Can families contact someone now to express their interest in being in the clinical trial?

Interested families should contact a clinical trial site that is currently enrolling to express their interest.

Additional information about the clinical trial and trial site(s) may be found here

Previous letters to the Rett syndrome community, including frequently asked questions from the community, can be viewed at here

There is a comprehensive travel and expense policy in place to cover trial-related costs and expenses for participating families.

Trial-related costs and expenses are paid by Neurogene; more details on the specific policy can be provided by the clinical trial site.

As with any clinical trial, participants may or may not benefit from this research. There are potential risks, and there is no guarantee that being in this study will help the participant

Sincerely,

Kimberly Trant, RN, MBA Executive Director Patient Advocacy and Engagement

www.neurogene.com

Click

Reverse Rett Key numbers

2010-2023

£1.39 million to the Gene Therapy Projects including Stuart Cobb (Neurogene) and Steve Gray (Taysha)

Let's not forget...

£1.3 million of funding to CIPP Rett Centre since 2019

An additional $673,009 to the Cobb Lab separate from Gene Therapy Consortium funding

9 years of funding

HealthTracker/REST questionnaire used every day by the CIPP Rett Centre to manage the health of complex patients with Rett

$611,438 to MECP2 Duplication Syndrome projects

$7,475,048

£1.65million to MECP2 Consortium (including Adrian Bird, Gail Mandel and Michael Greenberg) which led to Gene Therapy Consortium, current gene therapy programs and others in the pipeline to Rett Syndrome Research Trust research projects since 2010

These are big numbers. But all of it has been raised by families and communities of people with Rett the hard way.

We want to keep funding the CIPP Rett Centre and ongoing RSRT research programs which will lead to improved gene therapies and cast a cure for Rett in stone regardless of what happens with current programs. But we can't do it without your help.

Where does our regular income come from at Reverse Rett?

95%

Did you know that of our funding at Reverse Rett comes from our brilliant community fundraisers with only 5% regular income from our monthly donors and 365 Club members?

This means we have to raise more than...

£700,000

...a year to continue to do all the vital work we do to deliver treatments for Rett Syndrome.

This includes:

Funding the CIPP Rett Centre

Running the Rett Registry UK

Providing clinical trial support services including recruitment and logistics for families

Working with pharma to facilitate UK clinical trials

Outreach to find undiagnosed and unregistered patients of all ages

Preparation for health technology assessments for these emerging treatments

Providing critical health information for families so that our much loved people with Rett stay as healthy as possible until treatments are available

And more!

365 Club 1.6%

Regular Donations (JustGiving + Gift Aid) 1%

Regular Donations (Direct Debit) 2.6%

Shortfall (94.8%)

84 £2450

Regular monthly donors totalling

199 £995

365Clubmembers at£5amonth totalling

Click below to join!

Click below to become a monthly donor

Does your child have a positive MECP2 mutation?

With gene therapy clinical trials approaching, it's more important than ever that we are able to accurately state the number of UK children, young people and adults with a confirmed genetic mutation or deletion on the MECP2 gene.

This is because the gene therapies which are currently being trialed are specifically targeting the MECP2 gene. All patients must have a confirmed genetic mutation to take part and, should the trials show the therapies to be effective, a confirmed genetic mutation will be part of the eligibility process for any approved treatments which become available thereafter. We are currently sending out call outs for expressions of interest in potential UK gene therapy trials to help demonstrate need and interest in the UK.

When we set out to contact families of registered patients, we are only contacting families of patients with a confirmed genetic mutation Therefore, if you know your child does have a positive mutation, but you do not remember if you marked this on the Rett Registry UK, you should check this asap and mark the mutation as positive

If your child does not have a confirmed mutation or if you don't know if they do or not, please email rachael@reverserett.org.uk and we will help you find out.

If you cannot get into the registry for any reason, please email ewelina@reverserett.org.uk and she will help you.

www.rettregistry.co.uk

Call for expressions of interest

When to expect your EOI email:

Born in 2019 to present day - email due on or before 24 06 23

Born between 2014-2018 - email due on or before 04.07.23

Born before 2014 but after 2006 - email due on or before 07.07.23

The reason we are checking all age groups is:

Because we do not yet have confirmation of the age groups

Because protocol is sometimes amended part way through a trial to include patients at either end of the age range.

Families of people born before 2006, we are going to be emailing you as well, just so that we have the numbers to show to companies to say, whatever next for adults, we are warm here for an adults’ trial or better still, for approved therapies for our much beloved adults with Rett.

If you have not marked the patient's record with a positive MECP2 mutation, you will not receive this email. If you know your person has a mutation but you don't know what it is, please sign into the registry and make sure you have at least marked down that the test result was positive. We are only able to contact MECP2 positive patients for gene therapy trials.

If you need any help at all, either with accessing your registry account or deciphering genetic results, do get in touch using info@reverserett.org.uk or through any of the socials @reverserett.

URGENT APPEAL

This is an urgent appeal for funding to keep the CIPP Rett Centre, a specialist treatment and research centre for people with Rett Syndrome going.

The CIPP Rett Centre manages the complex care of patients OF ALL AGES and conducts research including clinical trials

There are no approved treatments for Rett Syndrome BUT if carefully managed, children with Rett Syndrome can live into adulthood and beyond

The CIPP Rett Centre is currently 100% funded by Reverse Rett and receives no NHS funding.

The reality is that many UK children, young people and adults with Rett do not have access to doctors who understand the condition or who have experience in managing the complex health of people with Rett Syndrome.

This is not the case for patients who attend or are monitored by Professor Santosh and his team at CIPP. Currently the team at CIPP, which is based at King's College Hospital in London are monitoring more than 150 complex UK patients with Rett Syndrome

This funding is what enables children, young people and adults with Rett Syndrome, who have a clinical need, to have their health care managed by the Centre at no cost to them, without delay.

But just one year of funding for the CIPP Rett Centre costs £338,000.

Professor Santosh and his team at the CIPP Rett Centre made a plan personalised to Lauren’s complexities. This has already proved invaluable for someone with the combination and volume of different medicines that she takes.

The team were also brilliant throughout lockdown, checking in regularly to see how Lauren was and tweaking her medications if necessary. They are also on hand to answer questions and support us. The CIPP Rett Centre has really made a huge difference to our lives.

- Helen, Mum to Lauren

100% OF ANY DONATION OR FUNDRAISING PAGE MADE THROUGH THIS CAMPAIGN WILL GO DIRECTLY TO THE CIPP RETT CENTRE

There are four ways you can help:

SET UP YOUR OWN JUST GIVING PAGE

Set up a Just Giving page specifically for the centre and take on a challenge or hold an event. Let everyone know that 100% of their donation plus gift aid will go to the CIPP Rett Centre.

Fundraising for the first time can be daunting but we are here to help you and will help you with every aspect to ensure that you can maximise your donations.

www.justgiving.com/reverserett

DONATE TO THIS CAMPAIGN

That will ensure that 100% of your donation plus gift aid will go towards funding the centre.

We know that we are living in difficult times and not everyone has the time to train and take on a challenge or make a donation so you can help us by sharing across your personal network.

SHARE WHAT YOU ARE DOING

Email friends who may be in a position to help, ask your employer if they support charities and make your case for Reverse Rett.

Share our posts on your social media pages. Sometimes support comes from the unlikeliest of places so let everyone know how important Reverse Rett and the CIPP Rett Centre are to you.

DONATE YOUR BIRTHDAY

Donating your birthday on Facebook/Instagram is a simple but extremely effective way to raise money. CLICK HERE to find out how.

The focus of the CIPP Rett Centre is to be a beacon of care and support for patients with Rett Syndrome all across the UK.

We strive for clinical excellence; by harnessing a translational approach, we will continue to impact the lives not only of the individual patients and families we care for but also, of the wider Rett community.

We are very grateful to Reverse Rett and all their supporters for making this work possible.

- Professor Paramala Santosh

Jurassic Juggernauts

By Andy Stevenson

In May I was part of a 14 strong team of men who took on the Jurassic Coast Ultra Challenge. The Jurassic Juggernauts walked 100k in Dorset raising £47,697.66 in the process which exceeded all our expectations.

After months of long walks through the winter we all felt as prepared as we could be for what lay ahead. None of us had ever walked that far before so we were all a bit nervous when we met up for dinner the night before Six of us were dads of a child with Rett Syndrome ranging from 3 to 32, two were brothers and the rest were friends and hardcore supporters of Reverse Rett. Most of us were sensible as we had an early start the next morning but some were led astray by Robbie Anderson and got stuck into the Guinness.

We set off in good spirits in lovely weather and soon broke off into groups On a walk that long people need to move at their own pace but we met up at each rest stop to check on each other and left at the same time as a team.

As the day progressed and we hit the giant hills along the coastal path the talking stopped as we were all in our own little bubble of discomfort. It was baking hot and many of the walkers taking part were struggling, once we’d hit the 50k point we all got a boost as it felt that we were now mentally counting down rather than up. Dinner was at 58k and it was a chance to eat some hot food, change clothes and generally take a little bit longer to rest and recharge the batteries As five of the team were from Dorset they managed to persuade their wives, partners and one mother-in-law to support us by bringing fresh clothes to the halfway point and giving us lifts to the start and picking us up from the finish. This made a huge difference and it was a real boost to see Hannah, whose dad and brothers were part of the team at the dinner check point. It reminded all of us why we were putting ourselves through this grinder

It was then headtorches on and off for the final 42k. There’s not much to say about the next 30k in the pitch darkness It was pretty miserable and much of it was spent looking down at the floor to ensure there were no accidents. We got some funny looks walking through Weymouth and also a £20 donation from a dog walker who asked who we were walking for.

I explained about Rett and how myself and Ross (we were walking together at this point) had daughters with the condition It might have been tears in my eyes but it looked like they were in his as he took his wallet out.

The jokes then started to flow in an effort to keep spirits high They were all pretty forgettable except for the monkeys one, thank you, Mark Walls. The banter through those hours was great and helped all of us keep moving despite the blisters and other injuries

By the team we all reached the 82k rest stop it was looking like a casualty ward. All of us were struggling in some shape or form and there were a lot of grumpy people sitting about wrapped up in tin foil. The important thing was that we were still going and determined to get to the end.

When the team came together we had vowed to start together and finish together. That is easier said than done over that sort of distance. By the time we got to the last 5k I was limping along at the back with rowing legend, Ross. Then at 99k Scott and Simon were waiting to walk the last kilometre with us. As we turned the corner to the finish the rest of the team appeared from nowhere and we all walked across the line together. It was emotional to say the least.

I’ve lost count of the challenges I’ve done since being introduced to Rett Syndrome in 2002. One thing is for sure though this one was truly unforgettable, not because it was tough, they are all tough in different ways or the scenery though it was spectacular but because of the people.

Everybody got on well, not just on the challenge but also on the WhatsApp group in the months leading up and the weeks after, it was a real privilege to spend time with people who hate Rett as much as I do and who are happy to go that extra kilometre to help make it extinct

After we finished and were tucking into breakfast we met 2 year old, Edith who was only diagnosed a few months ago Her family came to see us at the finish and it reminded us all (as if we needed it) why we were putting our bodies through the wringer.

Huge thanks to those in the team who don’t have a family member with Rett Syndrome. You guys don’t have to do stuff like this, you don’t see the struggles our children go through each and every day but you stand with us and do it anyway The aussies have a word for that....Mateship!

A final thank you to the girls who kept us going, Hannah, Amber, Beth, Eliza, Kim, Mollie and Alba They are the ones living with Rett Syndrome, not us, one day you will have the life you deserve.

Mollie’s dad, Mark Walls who travelled from Northern Ireland to join the team summed it up perfectly.

“We got everyone through the 100km challenge based on heart, desire and a common goal To get rid of Rett!

Great friendships were formed and a camaraderie was built out of love for our children regardless of our background, social class or beliefs

We talked openly, shared experiences, asked questions and all in the name of ridding ourselves of Rett At this pivotal time whilst my heart aches, I’m truly buoyed by the desire and determination shown to get rid of Rett.

To those who have suffered for so long, I salute you and admire your courage as torchbearers to keep going to get us to the stage we are at now.”

Why do we

to do so much fundraising at Reverse Rett?

By Andy Stevenson

As you’ll see from this edition of the newsletter, there is always a lot of fundraising going on at Reverse Rett, whether it’s the Annual London Gala, the Big Give Christmas Challenge or team challenges like the Action Challenge events, the Great North Run, Royal Parks Marathon, Kilimanjaro etc.

We often hear people saying that it looks like we raise a lot of money and we do, but we do a lot with the money we raise too.

Since the pandemic, fundraising has become more and more difficult. People are giving less to charities across the board because of the combined effects of the cost-of-living crisis, war in Ukraine, increase in energy bills and more.

This means we have to run more events, campaigns and challenges to generate the same income with less resource!

There has never been a better time for children, young people and adults with Rett Syndrome and I wake up every day excited to push forward with this work There are so many vital aspects of delivering treatments for Rett.

We can’t hold back on any of it. But we need your help to support our work.

need

Reverse Rett Lakers

By Andy Stevenson

The Reverse Rett Lakers took on a beast of a challenge on the weekend of June 10th. Despite sweltering in the heat the five strong team made it safely to the finish line in Kendal with 100k under their belts.

Bonnie McKendrick was walking for her daughter, Sophie and Alba’s Amblers consisted of her parents, Dan Thorne and Megan Shepherd plus Dan’s brother, Ben and friend Rupert Hodges

Both Sophie and Alba and their families were at the finish line to greet the walkers which made the event so special for everyone. Despite the blisters, the oppressive heat and various injuries those two little girls made the team dig very deep to ensure they got to the end.

They may have been a small group but they punched well above their weight as they raised a whopping....

£21,744.53

‘It was brilliant to be able to be in Kendal to cheer the Reverse Rett Lakers in from their epic challenge. We found it incredibly moving to see Sophie, Harry and Georgia greet Bonnie in their ‘we love you mum’ t-shirts and to see little Alba join her exhausted parents to walk their last few steps with them over the finish line. As always, we are deeply grateful to and humbled by the sight of dedicated families like these in a Reverse Rett tshirt giving their all to support vital work to accelerate treatments for our children and theirs. Thank you so much to all of you who took part.’

- Rachael Stevenson, Co-Founder, CEO Reverse Rett

Meet our new Operations Manager

Reverse Rett: small team, big job. Meet the new Operations Manager who will help us move forward to meet the challenges ahead full throttle.

After a thorough recruitment process which took several months and many layers of due diligence, we are delighted to share that Catherine Coles has taken the role of Operations Manager at Reverse Rett.

Catherine is no stranger to Reverse Rett, having been involved in fundraising and volunteering since the organisation was founded in 2010. Whilst the standard of applicants was extremely high, Catherine's operational experience combined with two decades of operations experience and team building acumen made her the stand out candidate for the role. Catherine also has a daughter, 27 year old Georgina who has Rett Syndrome.

I am delighted to introduce myself as the new Operations Manager at Reverse Rett.

I have been involved with Reverse Rett from the beginning in 2010 and have participated in quite a few challenges over the past 13 years and volunteered at the London Gala many times.

. My daughter, Georgina, is twenty-seven and has Rett Syndrome. Having the opportunity to play even a small part in bringing disease modifying treatment to her and all the people living with Rett Syndrome is a dream come true and one I will be immensely proud of.

I have 20+ years of organizational skills I am bringing to this role, managing multiple projects for different stakeholders, producing reports with tight deadlines while keeping a can-do positive attitude.

I have excellent team leading skills, resulting in my colleagues being confident in bringing issues to me for assistance in finding resolutions to complex situations. I bring an open, kind balance approach to being a leader, I enjoy building strong positive relationships as I believe this brings out the best in people.

I am thrilled to be joining what is already a strong team, albeit small and working together to make the ultimate difference for our children, young people and adults with Rett Syndrome.

- Catherine Coles, Operations Manager

catherine@reverserett.org.uk

CatherinewithGerogina

Sea to Summit

In October, seven women from North Berwick will climb Africa's highest mountain, Mt Kilimanjaro for Reverse Rett.

The Sea to Summit team were inspired by Eliza, the daughter of Reverse Rett trustee, Catherine McKinney.

They have already started their training and have taken on some of the UK's toughest climbs already in their quest to be ready.

They have also started fundraising by holding a coffee morning, having an open gardens afternoon in the local area and Altitude, a night of tunes and dancing at the Glen Golf Club.

As always, with this being North Berwick, they've had fantastic support from the local community with a deluge of prizes being donated for various auctions and raffles.

You can follow the team's progress as they continue their preparations for Kili by liking their Facebook page HERE

CatherinewithEliza

Do you live in or around London or the South East?

If so, we need your help with the Annual London Gala.

The Gala is our biggest fundraising event of the year and we generate much of the income from auction and raffle prizes and people attending on the night!

Please think:

Do you or anyone else you know have a holiday home in an interesting location?

Do you know of any local businesses that would be willing to help out by donating an item/s for the Auction, raffle or goodie bags?

Can you attend the gala? Can you get a table together?

Hazel is a Fundraising Associate here at Reverse Rett as well as grandmother to a remarkable young person with Rett

Over the years Hazel has garnered many prizes for the annual gala, from Freddie Mercury signed books to Louis Tomlinson VIP tickets,

Please contact Hazel at hazel@reverserett.org.uk if you think you can help.

The Lives of Reilly

Mark 'Mavis' Reilly has been taking on challenges for Reverse Rett since his daughter, Dionne was diagnosed with Rett Syndrome.

Mavis played for Kilmarnock for 11 years and winning the Scottish Cup in 1997. He also had spells at Motherwell, St Mirren and St Johnstone in a stellar footballing career.

The Lives of Reilly is an inspiring read which takes in Mavis's football career, his inspiration, Dionne and his numerous challenges for Reverse Rett, focusing on his trip into the Sahara desert to take on the world's toughest foot race, the Marathon des Sables.

All proceeds from the book will go to Reverse Rett and you can buy your copy below for just £10

MarkwithDionne

It's an honour and a privilege to write the foreword for The Lives of Reilly, a book that's poignant and inspiring as it is entertaining. Enjoy the journey!

The Lives of Reilly is an astonishing account of an amazing man on an epic journey of super-heroic proportions. Just reading about this honourable man will make you a better person.

- Ally McCoist, Rangers and Scotland Legend.

- Pat Nevin, Broadcaster, pundit and ex-professional footballer

Father's Day

On Father's Day, we posted asking families to share pictures of their much loved family member with Rett.

Here are some of the pictures that came back to us:

Meet the...

A group of fearless individuals driven by a shared mission to make a positive impact. They are setting off on a remarkable journey across Iceland to raise money for Rett Syndrome.

I am Louise from Loughborough I’m doing the Ice and Fire Trek because of our daughter Georgia, who deserves the best from this world and the chance at improving her life. I’m really excited about the trek and spending time with everyone, many of whom I've not met before with the solidarity of our shared challenge

Hi, I'm Emma from South Wales. I'm doing the Fire and Ice trek in memory of my beautiful Daisy She deserved to live a full life I hope, by participating in this challenge, we can make ground breaking treatments and therapies a reality for those with Rett and for future generations. I am looking forward to meeting some very special people on this journey.

Hi! I’m Jo from Switzerland. I’m trekking up volcanic scree and wading through icy rivers for my daughter Klara - she’s my inspiration! And with the crazy, courageous bunch of women on our team, together we’ll reach the summit and help improve the lives of everyone with Rett Syndrome. I can’t wait to stand on top and see that view!

I’m Chloe and I’d never heard of Rett Syndrome before I met Rachael We met by chance and now I’m so looking forward to doing the Fire and Ice Trek alongside her.

While a little nervous about the training, the trek, the terrain, the camping… I’m excited too - about a first trip to Iceland, but also about meeting the team, new friends, and of course about raising money for Reverse Rett!

Jo & Klara

Emma

Louise&Georgia

Chloe

Rachael &Amber

I’m doing the trek in honour of our Amber, Beth and everyone affected by Rett Syndrome. I long for the day when I see our UK children, young people and adults with Rett able to access emerging treatments that will significantly improve their lives. Bring on the volcano!

Hey there! I’m Caroline, mother, polyglot and Early Years Lead in a North London infant school. Willing explorer, I am looking forward to experiencing the long hours of daylight and sighting a whale. I am nervous my joints mightn’t last out and what to do about this

I am Catherine , I live in Spain, I am a mother of four daughters, my youngest Georgina has Rett Syndrome, I am doing the Fire and Ice Challenge to raise funds for Reverse Rett. I am excited about the trip, sharing the challenge with 10 other ladies all with the focus of making change happen for Georgina and her Rett sisters

I am Soraya a retired Doctor, with a keen supportive passion for cutting edge research on the verge of discovering treatments through various modalities, in the case of Rett, gene therapy .

I wanted to support this team having met the CEO, on my last fundraising mission in the Sahara last year

This challenge , and the money I am raising will get us closer to finding a way to modify Rett syndrome. Science is very close ,and I am embracing the challenge enthusiastically for this team doing something I love, hiking

I’m Amal and I’m doing this because I have some dear close family members who are suffering with Rett Syndrome and really special people in my family who are dedicated to Reverse Rett. I want to be part of facilitating the cures that my family are working so hard to bring about.

AmalwithAmber

I'm Wesley, the honorary male ice maiden. Having done the Saharan Challange in October, it's a privilege to be amongst such strong and fearless women again and raising awareness of Rett Syndrome in the land of fire and ice

Catherine&Georgin

Caroline

Soraya