Sarepta Therapeutics Announces Approval in Japan of ELEVIDYS, a Gene Therapy to Treat Duchenne Muscular Dystrophy
People aged 3 to under 8 who do not have deletions in exons 8 and/or 9 of the DMD gene and who test negative...
SonoThera Ultrasound-Mediated Delivery Method Can Deliver Full-Length Dystrophin to Cells
The findings of the study, which are currently being developed for SonoThera's Duchenne Muscular Dystrophy (DMD) treatment, demonstrate the capability of Ultrasound-Mediated Delivery technology...
Scribe Therapeutics and Prevail Therapeutics to Share In Vivo CRISPR-based Genome Editing Data at ASGCT 2025
Scribe Therapeutics, a genetic medicines company unlocking the potential of CRISPR to transform human health, today announced its participation in the 28th American Society...
Capricor Therapeutics Announces Completion of Mid-Cycle Review Meeting with FDA on Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy
Capricor Therapeutics , a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the completion of a...
Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy
Today, Dyne Therapeutics, a clinical-stage company dedicated to developing life-changing treatments for individuals with genetically driven neuromuscular diseases, announced that DYNE-251 has been designated...