- Positive 24-month data from ongoing Phase 2 TOPAZ trial extension supports potential benefit of apitegromab for patients with Types 2 and 3 Spinal Muscular Atrophy (SMA)- Actively enrolling in pivotal Phase 3 SAPPHIRE clinical trial for apitegromab- Discovery pipeline advancements made for LTBP-TGFβ1 target to tre.
Sarepta Therapeutics has more clinical data showing the safety and efficacy of its experimental gene therapy for Duchenne muscular dystrophy. A pivotal Phase 3 test is already underway and could post data next year but the company is also talking with regulators about the possibility of a submission under the accelerated approval pathway.
Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) ("Amylyx" or the "Company") today announced a poster on the ongoing international Phase 3 PHOENIX trial (study A35-004, NCT05021536) of AMX0035 (sodium phenylbutyrate
Sarepta Therapeutics (SRPT) Investigational Gene Therapy SRP-9001 for DMD Demonstrates Significant Functional Improvements Across Multiple Studies streetinsider.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from streetinsider.com Daily Mail and Mail on Sunday newspapers.