Physical activity and rehabilitation are key components of Duchenne muscular dystrophy treatment, despite recent drug therapy advances. "Rehabilitation care and rehabilitation teams with experience ...
– U.S. FDA has confirmed that screening and dosing may proceed in Study SRP-9005-101 for LGMD2C/R5 – Enrollment and dosing completed in Study SRP-9004-102 for LGMD2D/R3 – Data expected for SRP-9003 ...
BOULDER, Colo.--(BUSINESS WIRE)--Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food & Drug Administration (FDA) has ...
Both cycling and home-based exercise training helped maintain gait and balance parameters in children with Duchenne muscular dystrophy (DMD), with cycling training also improving antero-posterior ...
NASHVILLE, Tenn., Feb. 4, 2026 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company focused on developing new products for rare diseases, announced today ...
Atossa's Rare Pediatric Disease Designation for (Z)-endoxifen in neuromuscular diseases qualifies for a future PRV upon FDA approval SEATTLE, Feb. 5, 2026 /PRNewswire/ -- Atossa Therapeutics, Inc.
More than 30 rare muscular dystrophy types cause progressive muscle weakness from inherited gene mutations. Combined, they affect about 1 in 5,000 to 8,000 people. Common subtypes vary by age of onset ...
FDA and Capricor aligned on endpoints for HOPE-3 pivotal trial HOPE-3 pivotal trial completed; topline data expected mid-Q4 2025 to support BLA resubmission Company preparing to resubmit CRL response ...