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Session 4C: Clinical Advances in Oligonucleotide-Based Therapies for Neuromuscular Diseases
Session Chair(s)
Saraswathy V. Nochur, PhD, MSc
Chief Regulatory Officer
Alnylam Pharmaceuticals, United States
Several oligonucleotide-based therapeutic candidates have been evaluated in the clinic for the treatment of neuromuscular diseases, including two for rare diseases. This session will explore the development of 3 different molecules, one about to enter the clinic, another getting ready for NDA, and a third that has been approved by FDA and EMA. While each of the disease states for these agents are distinct, there are similarities in the clinical development strategies and the challenges faced. Clinical data will be shared.
Learning Objective :
Speaker(s)
Session Co-Chair
Lois Freed, PhD
FDA, United States
Director, Division of Pharmacology/Toxicology-Neuroscience (DPT-N), CDER
Development and Approval of Nusinersen for Spinal Muscular Atrophy: Challenges and Successful Strategies
Wildon Farwell, MD
Biogen , United States
Senior Director, Clinical Development
Hereditary ATTR Amyloidosis: Long-Term Clinical Experience with Patisiran, an Ivestigational RNAi Therapeutic, in an Open Label Study
John L. Berk, MD
Boston University, United States
Associate Professor of Medicine, School of Medicine
Development of Ionis-MAPTRx, the First Tau-Lowering Antisense Oligonucleotide, in Patients with Mild AD
Laurence Mignon, PhD
Ionis Pharmaceuticals, Inc., United States
Director, Clinical Development
Panel Discussion
All Session Speakers, United States