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...
Avidity Biosciences Announces U.S. Managed Access Program (MAP) for Investigational Therapy del-zota (Exon 44 Skipping)
Avidity Biosciences announced its Managed Access Program (MAP) for investigational therapy delpacibart zotadirsen (del-zota) for eligible people with Duchenne muscular dystrophy mutations amenable to...
Potential of SAT-3247 to Restore Muscle Regeneration in Duchenne Published in Nature
Satellos announced the publication in Nature Communications of new research from a scientific team at the Ottawa Hospital Research Institute (OHRI) that validates the...