Wednesday 7th December 2022
18.00pm – 20.30pm: Early Registration & Exhibition
Thursday 8th December 2022
8.00am – 8.45am – Registration
8.45am – 8.50am – Welcome, Jim Dowling, TREAT-NMD Chair
8.50am – 9.00am: Introduction & TREAT-NMD Updates, David Allison, CEO, TREAT-NMD
SESSION ONE: Patient engagement
9.00am – 9.20am: Established groups, Erin Ward, President of MTM-CNM Family Connection
9.20am – 9.40am: Emerging groups, Terry Pirovolakis, founder of CureSPG50
9.40am – 10.00am: Emerging Groups, Rick Ferguson, CEO of the Daniel Ferguson Foundation and Dr Michelle Lorentzos, paediatric neurologist and Clinical Trials Medical Lead at The Children’s Hospital, Sydney, Australia
10.00am– 10.20am: Panel discussion & questions
10.20am – 10.50am: Refreshments, Exhibition & Posters
SESSION TWO: Clinical trial considerations in ultra-rare patient groups
10.50am – 11.10am: Patient Advocacy, Sharon Hesterlee, MDA
11.10am – 11.30am: Industry Perspective, Tristen Moors, Vice President, Clinical Operations at Glycomine, Inc
11.30am – 11.50am: Patient Identification, Chris Weihl, Professor of Neurology and Head of the Neuromuscular Section at Washington University School of Medicine in St. Louis, Missouri
11.50am – 12.10pm: Panel discussion & questions
Symposia
12.10pm – 12.30pm: Symposia 1 – Sarepta: Community and Collaboration
Accelerating Research and Drug Development
in Limb-Girdle Muscular Dystrophy, André Müller-York, MD,
LGMD Medical Lead and Global Medical Affairs at Sarepta Therapeutics Ltd.
12.30pm – 12.45pm: Symposia 2 – Roche: Equity, diversity & inclusion in DMD, Prof. Volker Straub, Director of John Walton Muscular Dystrophy Research Centre, Newcastle University, UK
12.45pm – 13.00pm: Symposia 3 – Ultragenyx: Long chain fatty acid oxidation defects (LC-FAODs): a group of rare disorders with a neuromuscular presentation, Shailly Jain, MD, FRCPC, FCCMG, Clinical Geneticist & Metabolics Specialist and Associate Professor, Edmonton Medical Genetics Clinic, University of Alberta.
13:00pm-13:10pm: Symposia 4 – Biogen: Spinal Muscular Atrophy Disease Registries: Overview and Recent Progress, Stephanie Raynaud, MD, Global Associate Medical Director, Biogen
13.10pm – 13:55pm: Lunch, Exhibition & Posters
SESSION THREE: Global Neuromuscular Care and Access to Treatment
13.55pm – 14.15pm: Family/patient advocate: Treatment access in emerging markets, Vanina Sanchez, mother of Joaquín, who has Spinal Muscular Atrophy type II
14.15pm – 14.35pm: The clinician’s perspective: Providing guidance and care in an era of commercially available treatment(s), but with limited access, Edoardo Malfatti, Centre de Référence de Maladies Neuromusculaires
14.35pm – 14.55pm: Patient access solutions that address outcome certainty, affordability, value recognition and capacity: Industry Perspective, Julian Nam, Global Access Strategy Leader, F. Hoffmann-La Roche Ltd
14.55pm – 15.15pm: Rolling our treatments globally, Nicole Mittmann, Chief Scientist and Vice-President of Evidence Standards, CADTH
15.15pm – 15.35pm: Panel discussion & questions
15.35pm – 16.00pm: Refreshments, Exhibition & Posters
SESSION FOUR: Living with NMD: practical considerations and QOL
16.00pm – 16.20pm: Nutrition & weight loss, Zoe Davidson, Advanced Accredited Practising Dietitian specialising in paediatric nutrition
16.20pm – 16.40pm: Exercise, Gita Ramdharry, Consultant Allied Health Professional in Neuromuscular Diseases at the Queen Square/ MRC Centre for Neuromuscular Diseases, National Hospital for Neurology and Neurosurgery, UCLH
16.40pm – 17.00pm: Respiratory considerations, Robert Graham, Pediatric critical care physician & Senior Associate in Division of Critical Care Medicine, Boston Children’s Hospital
17.00pm – 17.20pm: Panel discussion & questions
19.00pm: Networking Event
19.45pm: Keynote speaker, Annemieke Aartsma-Rus, PhD, Professor of Translational Genetics, Department of Human Genetics, Leiden University Medical Center
Friday 9th December 2022
8.00am – 9.00am: Welcome & Refreshments
SESSION FIVE: COVID impact on Outcomes, Care & Clinical Trial Design
8.30am – 9.00am: Covid Sequelae, Maxwell Damian, Department of Neurology, Addenbrooke’s Hospital, Cambridge University Hospitals
9.00am – 9.30am: Impact of COVID on clinical trial design & outcomes, Teji Singh, Sarepta
9.30am – 9.50am: Remote Outcomes, Meredith James, Clinical Specialist Neuromuscular Physiotherapist at the John Walton Muscular Dystrophy Research Centre at Newcastle, United Kingdom
9.50am – 10.20am: Biopsychosocial consequences of the COVID-19 pandemic for people with neuromuscular diseases, Lone Knudsen, Clinical research, National Rehabilitation Center for Neuromuscular Diseases, Aarhus, Denmark
10.20am – 10.40am: Panel discussion & questions
10.40am – 11.25am: Refreshments, Exhibition & Posters
SESSION SIX: Emerging Therapeutics options in NMD
11.25am – 11.45am: Pre-clinical and clinical CRISPR programs, Sean Burns, VP Disease Biology, Intellia Therapeutics
11.45am – 12.05pm: Gene Therapy, Carsten Bonnemann, Senior Investigator, Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS
12.05pm – 12.25pm: N-of-1 therapies for patients with very rare mutations, Julia Vitarello
12.25pm – 12.45pm: Panel discussion & questions
Symposia
13.00pm – 13.15pm: Symposia 5 – Novartis: Living with Spinal Muscular Atrophy and the Evolving Treatment Journey, Dr. Kathryn Selby, Investigator at BC Children’s Hospital Research Institute
13.15pm – 13.25pm: Symposia 6 – Project Hercules, Duchenne UK: Evolving clinical practice: what can we learn from Project HERCULES?, Josie Godfrey, Strategic Director for Project HERCULES and Prof. Volker Straub, Director of John Walton Muscular Dystrophy Research Centre, Newcastle University, UK
13.25pm – 13.35pm: Symposia 7 – PTC Therapeutics: From clinical trials to real-world evidence: updates on the management of nmDMD, Dr. Christian Werner, Executive Director, Global Medical Affairs – Global DMD Lead at PTC Therapeutics
13.35pm – 14.35pm: Lunch, Exhibition & Posters
SESSION SEVEN: Preparing for future trials and interventions
14.35pm – 14.55pm: Newborn/neonatal screen, Pranesh Chakraborty, Medical and Laboratory Director for Newborn Screening Ontario
14.55pm – 15.15pm: Care 4 rare/obtaining a diagnosis, Kym Boycott, Professor of Pediatrics at the University of Ottawa in Canada
15.15pm – 15.35pm: Development of a complex drugs program, Julie Parsons, Children’s Hospital Colorado
15.35pm – 15.55pm: Panel discussion & questions
16.25pm – 16.35pm: Conference Close and Presentations
For announcements, see the TREAT-NMD Newsletter, sign up here:
Venue
Vancouver Convention Centre
1055 Canada Pl,
Vancouver,
British Columbia
V6C 0C3,
Canada
