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Session 1C (Clinical Development Track): Neuromuscular
Session Chair(s)
Lois M. Freed, PhD
Director, Division of Pharmacology/Toxicology-Neuroscience (DPT-N), CDER
FDA, United States
Stephen B. Shrewsbury, MD, FFPM
Advisory Board
LifeSplice Pharma, Canada
This session will review the encouraging emerging clinical data generated over the last 18 months with therapeutic oligonucleotides in neuromuscular disease, where splice switching using the alternative splicing pathway is emerging as a natural process to be harnassed by modern medicines.
Speaker(s)
Antisense Therapies for Neurodegenerative Diseases
Richard Stephen Geary, PhD
Ionis Pharmaceuticals, Inc., United States
Senior Vice President, Drug Development
Update on Sarepta’s AVI-4658 in Exon 51 Skipping Amenable Duchenne Muscular Dystrophy Boys
Jerry R Mendell, MD
Nationwide Children's Hospital, United States
Professor of Pediatrics, Neurology, Pathology & Physiology
Update on GSK’s Drisapersen in Exon 51 Skipping Amenable DMD
Craig McDonald, MD
UC Davis Children's Hospital, United States
Update on Prosensa’s other Splice Switching Oligomers (PRO044) in DMD
Giles Campion, MD, PhD
BioMarin Nederland BV, Netherlands
Chief Medical Officer
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