WMS 2022 Industry Partners - October

WELCOME

Dear members of the World Muscle Society, friends and colleagues,

Welcome to the 27th annual congress of the World Muscle Society in Halifax.

This is a very special congress for me, as it is the first in-person congress while in post as President of the Society.

The last two congresses, while successful, felt extremely different from the traditional format of WMS congresses.

Many of us will have missed the lively discussions at the poster boards, informal networking at coffee breaks, the opportunity to make new friendships and refresh old ones, and being immersed for 4 days in the latest scientific and clinical developments in the neuromuscular field. Nevertheless, COVID has changed the world and the WMS had to adapt.

For our first congress in Canada, we decided to offer a hybrid format that would allow colleagues around the world to either join us in person or online. It is great to see more than 1,000 participants did make use of this offer, with a majority choosing to travel to Halifax.

On behalf of the board, our thanks go to our sponsors and exhibitors for their support in enabling this congress to go ahead, as well as our local organising committee and management team for their hard work behind the scenes.

I’m very much looking forward to either interacting with you virtually or to seeing you in Nova Scotia!

With very best wishes,

Volker Straub

President of the WMS

Argyle

WMS

HYBRID

INDUSTRY SYMPOSIA SCHEDULE

Ballroom

Day 2 - Wednesday 12th October

07:00-08:30

WMS Industry Symposium 1

The Era of Gene Therapy: Growing Evidence of The Clinical Reality

Argyle

WMS Industry Symposium 2 Shared decision-making in the management of late-onset Pompe disease

17:30-19:00

WMS Industry Symposium 3

Connecting the Dots: Charting New Frontiers in Duchenne

WMS Industry Symposium 4 Measuring Progression in FSHD: Implications for Clinical Trials

Day 3 - Thursday 13th October

07:30-09:00

WMS Industry Symposium 5

Moving the needle: improving outcomes in paediatric SMA and DMD

WMS Industry Symposium 6 Rethinking the science of SMA

13:30-15:00

WMS Industry Symposium 8

Navigating the changing disease landscape: continuity of care in Duchenne muscular dystrophy

07:00-08:30

Day 4 - Friday 14th October

WMS Industry Symposium 7

Targeting Fast Muscle Myosin: A Novel Approach to Protecting Muscle in the Dystrophinopathies

Please see: https://www.wms2022.com/ page/industry-symposia for other information.

All times Atlantic Daylight Time (ADT) - Local time in Halifax, Canada

THE ERA OF GENE THERAPY: GROWING EVIDENCE AND CLINICAL REALITY OF TREATING SMA

Wednesday October 12, 2022, 7:00 – 8:30 AM ADT

Room: Ballroom

Halifax Convention Centre

Halifax, Nova Scotia, Canada

AGENDA

7:00 7:05 AM

WELCOME & INTRODUCTION

Dr. Hernan Gonorazky, M.D. (Chair), Assistant Professor and Director, Faculty of Medicine, The Hospital for Sick Children, Division of Neurology and Neuromuscular Fellowship Program, University of Toronto, Toronto, Canada

7:05 7:20 AM

OVERCOMING HURDLES OF GENE THERAPY

Dr. Hernan Gonorazky, M.D.

7:20 – 7:45 AM

GENE THERAPY: THE CLINICAL REALITY

Prof. Francesco Muntoni, M.D., Director and Co-Director, Dubowitz Neuromuscular Centre and MRC Centre for Neuromuscular Diseases, UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children (GOSH) Foundation Trust, London, UK

7:45

8:15

BULBAR FUNCTION: GENE THERAPY IN PRACTICE

Dr. Giovanni Baranello, M.D., Clinical Associate Professor, Paediatric Neurology/Neuromuscular Disorders, Department of Developmental Neurosciences, UCL GOS Institute of Child Health, London, UK Nicole LaMarca, D.N.P., M.S.N., C.P.N.P., P.M.H.S., Global Medical Director, Data Exploration and Generation Lead SME, Global Medical A airs, Novartis Gene Therapies, USA

8:15

8:30

PANEL DISCUSSION AND CLOSING REMARKS

Discussion moderated by Nicole LaMarca, D.N.P., M.S.N., C.P.N.P., P.M.H.S.

We look forward to welcoming you to an informative and engaging event.

This event is supported, in part, by funding from industry. All support is managed in strict accordance with CME/CPD accreditation criteria and standards for commercial support. Industry Sponsored Symposia are organised by industry and not included in the main event CME/CPD credit o ering.

This meeting is sponsored and organized by Novartis Gene Therapies.

2022 Novartis Gene Therapies, Inc.

Speakers

Moderator

Louise Rodino-Klapac, PhD Executive Vice President

of R&D, Chief Scienti

Sarepta Therapeutics,

Ohio, USA

Emma Ciafaloni, MD, FAAN Co-Director, MDA Neuromuscular Clinic Professor of Neurology and Paediatrics University of Rochester New York, USA

Craig M. McDonald, MD

Department of Physical Medicine & Rehabilitation, Department of Paediatrics Director, MDA Neuromuscular Disease Clinics University of California Davis Health California, USA

Francesco Muntoni, FRCPCH, FMedSci

Director, Dubowitz Neuromuscular Centre Co-Director, MRC Centre for Neuromuscular Diseases

Great Ormond Street Institute of Child Health & Great Ormond Street Hospital for Children

Foundation Trust London, UK

SAREPTA

Logo

trademarks of Sarepta Therapeutics,

may

Measuring Progression in

FSHD: Implications for Clinical Trials

Wednesday, October 12, 2022

17:30 - 18:50 ADT / 16:30 - 17:50 EDT

Halifax Convention Centre: Argyle

Speakers:

Judith Dunn, Ph.D. President, Research and Development Fulcrum Therapeutics

Sabrina Sacconi, M.D., Ph.D.

Head of the Peripheral Neurology and Muscle Unit Centre Hospitalier Universitaire de Nice

Leo Wang, M.D., Ph.D.

Assistant Professor of Neurology University of Washington www.fulcrumtx.com

Roche in neuromuscular conditions – translating science into meaningful outcomes, together

Roche at the 27th International Hybrid Annual Congress of the World Muscle Society (WMS), 11–15 October 2022, Halifax, Canada

Join us in person or online to discuss the latest evidence and clinical perspectives on the management of neuromuscular diseases, including spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). We welcome you to our data presentations where we will be sharing insights and updates from our research programs. We also invite you to our satellite symposium*, where presenters will explore recent advances in the treatment of paediatric SMA and DMD. We look forward to seeing you at WMS!

Rethinking the

of

Dr Didu (Sandi) Kariyawasam

SMN-restoring therapies are transforming patient outcomes in SMA. However, variability in treatment response at the individual level remains striking. We explore what is known about the genesis, degeneration, and potential resurrection of the motor neuron in SMA, the host and intervention factors that may modify treatment response at the individual level, and the rationale for investigating strategies to optimize these responses.

who

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Targeting

Muscle

About the Symposium

The symposium will explore how targeting fast muscle myosin could potentially protect muscle in Duchenne and Becker muscular dystrophy.

The damaging consequences of muscle contraction without functional dystrophin will be reviewed by Dr. Lee Sweeney. When dystrophin is absent, muscle contraction generates sarcomeric stress, opening of membrane stress channels, and calcium in ux into the myo ber leading to myo ber degeneration with increases in circulating biomarkers of muscle damage.

Dr. John Vissing will discuss the impact of muscle contrac tion and exercise on biomarkers of fast muscle bre damage in dystrophinopathies and other muscular dystrophies.

Dr. Craig McDonald will discuss the clinical course of Becker muscular dystrophy and potential strategies for clinical trials.

Dr. Alan Russell will discuss identi cation of a fast myosin modulator to disconnect muscle injury from muscle contraction in dystrophinopathies to circumvent the struc tural stress caused by loss of dystrophin on DMD/BMD.

Lastly, Dr. Joanne Donovan will discuss most recent ndings clinical studies with EDG-5506 is a novel, rst-in-class, small molecule for the treatment of dystrophinopathies via this approach, which has potential either as a stand-alone therapy or in combination with dystrophin-targeted thera pies across the spectrum of dystrophinopathies as well as other muscular dystrophies.

After hearing from our speakers, we will open it up for Q&A from the audience.

ORDER OF SPEAKERS

"Preferential Fast Fiber Injury in Dystrophinopathies"

LEE SWEENEY, PH.D.

Department of Pharmacology & Therapeutics

FL

"Mechanical Stress Induced Injury: Changes in Biomarkers of Dystrophic Muscle during Exercise in LGMD and BMD"

JOHN VISSING, M.D., PH.D.

of Copenhagen Denmark

"Clinical Course of Dystrophinopathies"

CRAIG MCDONALD, M.D.

Davis Sacramento, CA

"Targeting Fast Myosin to Decouple Injury from Muscle Contraction in DMD and BMD"

RUSSELL, PH.D.

Scientific Officer Edgewise Therapeutics

"Clinical Development of EDG-5506 in BMD and DMD"

JOANNE DONOVAN, M.D., PH.D.

Medical Officer Edgewise Therapeutics

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At Amicus Therapeutics, we strive to fulfill the bold commitment we’ve made to the rare disease community. This commitment keeps us at the forefront of developing therapies for those who need them most.

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SEEKING TO IMPROVE LIVES THROUGH THE CURATIVE POTENTIAL OF GENE THERAPY

Astellas Gene Therapies is developing potential gene therapies for rare neuromuscular diseases

Please Join our Platform Presentation

O.06. Long term outcomes for X-Linked myotubular Myopathy (XLMTM) with gene replacement therapy, resamirigene bilparvovec: preliminary results from ASPIRO

Session: New Developments in Congenital Myopathies 2

Wednesday, October 12, 2022 11:00-13:00, Ballroom

Perry Shieh, MD, PhD

University of California, Los Angeles Los Angeles, CA

X-linked Myotubular Myopathy (XLMTM) is a rare, life-threatening monogenic congenital myopathy with an estimated incidence of 1 in 40,000-50,000 newborn males.1,2

1. Biancalana V, et al. Acta Neuropathol. 2017;134(6):889-904.

Graham RJ, et al. Arch Dis Child. 2020;105(4):332-8.

Inc.

TO LEARN

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