Avidity Biosciences Announces Positive Pre-BLA Meeting with U.S. FDA for del-zota in DMD44 with a Submission Planned for Q1 2026
Avidity Biosciences announced that the Company completed a positive pre-BLA meeting with the U.S. Food and Drug Administration (FDA) regarding its upcoming BLA submission...
Satellos Announces New Data Further Demonstrating Safety, Tolerability, and Functional Impact of SAT-3247 in First-in-Human Trial of Adults with Duchenne Muscular Dystrophy
Satellos Bioscience announced new data further demonstrating tolerability and initial efficacy of SAT-3247 in adults (aged 20-27 years) with Duchenne muscular dystrophy (Duchenne or...
Genethon Confirms 2-Year Efficacy in Duchenne Muscular Dystrophy Patients Treated With Its Low Dose Micro-Dystrophin Gene Therapy (GNT0004)
Genethon, released positive long-term efficacy data revealing three Duchene muscular dystrophy patients showed significant motor function gain as compared to untreated patients, and experienced...
Santhera Secures Agreement with Biomedica for the Distribution of AGAMREE (Vamorolone) in Russia
Santhera announces the signing of an exclusive agreement with Biomedica for the distribution of AGAMREE (vamorolone) in Russia, for the treatment of Duchenne muscular...
SonoThera Presents New Data at ESGCT 2025 Demonstrating Expression of Full-Length Human Dystrophin Using Targeted, Ultrasound-Mediated Delivery to Treat Duchenne Muscular Dystrophy
SonoThera, a biotechnology company dedicated to treating the root cause of human diseases by developing the next generation of genetic medicines, today announced it...