Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy
Atossa Therapeutics announced that the U.S. Food and Drug Administration ("FDA") has granted Rare Pediatric Disease ("RPD") designation to (Z)-Endoxifen for the treatment of...
Satellos Receives FDA and International Clearance to Commence Pediatric Phase 2 Testing of SAT-3247 for Duchenne Muscular Dystrophy
Satellos reported that it has obtained Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) and additional international regulators to...
Dyne Therapeutics to Apply for Accelerated Approval in Q2 2026 for Exon 51 Skipping Therapy (DYNE-251)
Dyne Therapeutics announced positive topline results from the Registrational Expansion Cohort (REC) of its Phase 1/2 DELIVER trial evaluating zeleciment rostudirsen (z-rostudirsen, also known...
Capricor Therapeutics Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy
HOPE-3 is a randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating Deramiocel in boys and young men with Duchenne muscular dystrophy. The study randomized...
ELEVIDYS Gene Therapy for Non-Ambulant Children with DMD: FDA OKs ENDEAVOR Cohort 8 Immunosuppression Study
Sarepta Therapeutics confirmed that the FDA has sanctioned the start of dosing in Cohort 8 of the ENDEAVOR (Study 9001-103) trial. This crucial cohort...