Wednesday 7th December 2022
6pm – 8.30pm: Early Registration & exhibition
Thursday 8th December 2022
8am: Registration
8.45am: Welcome
SESSION ONE: Patient engagement
9am: Established groups, speaker TBA
9.10am: Emerging groups, Terry Pirovolakis, founder of CureSPG50
9.30am: Emerging Groups, Nathan Peck, founder and CEO of Cure VCP Disease Inc
9.50am: Emerging Groups, speaker TBA
10.10am: Panel discussion & questions
10.30am: Refreshments & posters
SESSION TWO: Clinical trial considerations in ultra-rare patient groups
11.00am: Patient Advovacy, Jennifer Siedman, Director of Family Engagement for Courageous Parents Network (CPN)
11.20am: Industry Perspective, Tristen Moors, Vice President, Clinical Operations at Prothelia Incorporated
11.40am: Patient identification, Chris Weihl
12.00pm: Panel discussion & questions
12.20pm: Symposia 1 – Sarepta
12.40pm: Symposia 2 – Roche
12.55pm: Symposia 3 – Ultragenyx
13.10pm: Lunch & posters
SESSION THREE: Global Neuromuscular Care and Access to Treatment
14.10pm: Family/patient advocate: Treatment access in emerging markets, Vanina Sanchez, mother of Joaquín, who suffers from Spinal Muscular Atrophy type II
14.30pm: 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
14.50pm: Industry perspective, speaker TBC
15.10pm: Rolling our treatments globally, Nicole Mittman, Chief Scientist and Vice-President of Evidence Standards, CADTH
15.30pm: Panel discussion & questions
15.50pm: Refreshments & posters
SESSION FOUR: Living with NMD: practical considerations and QOL
16.20pm: Nutrition & weight loss, Zoe Davidson, Advanced Accredited Practising Dietitian specialising in paediatric nutrition
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
17.00pm: Respiratory considerations, Robert Graham, Pediatric critical care physician & Senior Associate in Division of Critical Care Medicine, Boston Children’s Hospital
17.20pm: Panel discussion & questions
19.00pm: Networking event
19.45pm: Keynote speaker, Geralyn Miller, Senior Director, AI for Good Research Lab
Friday 9th December 2022
8.30am: Welcome & refreshments
SESSION FIVE: COVID impact on Outcomes, Care & Clinical Trial Design
9.00am: Covid Sequelae, Maxwell Damian, Department of Neurology, Addenbrooke’s Hospital, Cambridge University Hospitals
9.30am: Impact of COVID on clinical trial design & outcomes, Teji Singh, Serepta
10.00am: Remote Outcomes, Meredith James, Clinical Specialist Neuromuscular Physiotherapist at the John Walton Muscular Dystrophy Research Centre at Newcastle, United Kingdom
10.20am: Psychological Impact of COVID, Lone Knudson, Clinical research, National Rehabilitation Center for Neuromuscular Diseases, Aarhus, Denmark
10.50am: Panel discussion & questions
11.10am: Refreshments & posters
SESSION SIX: Emerging Therapeutics options in NMD
11.40am: Genome Editing , Laura Sepp-Lorenizono, Chief Scientific Officer, Intellia Therapeutics
12pm: Gene Therapy, Carsten Bonnermann, Senior Investigator, Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS
12.20pm: N-of-1 therapies for patients with very rare mutations, Julia Vitarello
12.40pm: Panel discussion & questions
13.00pm: Symposia 4 – Novartis
13.15pm: Symposia 5 –TBC
13.25pm: Symposia 6 – Duchenne UK
13.35pm: Symposia 7 – PTC Bio
13.45pm: Lunch & posters
SESSION SEVEN: Preparing for future trials and interventions
14.45pm: Newborn/neonatal screen, Pranesh Chakraborty, Medical and Laboratory Director for Newborn Screening Ontario
15.05pm: Virtual trials, Yan Yiannakou, Clinical Director of the NIHR Patient Recruitment Centre, Newcastle UK
15.25pm: Care 4 rare/obtaining a diagnosis, Kym Boycott, Professor of Pediatrics at the University of Ottawa in Canada
15.45pm: Psychological impact, speaker TBC
16.05pm: Panel discussion & questions
16.25pm Close
For announcements, see the TREAT-NMD Newsletter, sign up here:
Venue
Vancouver Convention Centre
1055 Canada Pl,
Vancouver,
British Columbia
V6C 0C3,
Canada
