Nasdaq

FDA Approves Label Extension for Evrysdi® for Infants with Spinal Muscular Atrophy Under 2 Months Old

SOUTH PLAINFIELD, N.J., May 31, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has approved a label extension for Evrysdi® ...
Business Wire

Edgewise Therapeutics Announces Positive 4-Month Interim Results from the ARCH Open Label Study of EDG-5506 in Adults with Becker Muscular Dystrophy (BMD)

BOULDER, Colo.--(BUSINESS WIRE)--Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for the ...
Seeking Alpha

Capricor Therapeutics Announces Positive 3-Year Efficacy Results from HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy

*Baseline is referred to as start of HOPE-2 OLE study, changes in means are shown “I am extremely encouraged by these positive results from our HOPE-2 OLE study which continue to support the long-term ...
Seeking Alpha

Satellos Announces Encouraging Functional Data from the 28-day Phase 1b Open-Label Trial of SAT-3247 in Adults with Duchenne Muscular Dystrophy

Phase 1b data shows treatment with SAT-3247 was safe and well-tolerated Pharmacokinetic (PK) profile of SAT-3247 translated as expected to Duchenne Muscular Dystrophy (DMD) patients taking concurrent ...