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...
New Data Presented on Duvyzat (Givinostat) for Treatment of Duchenne Muscular Dystrophy at 2025 World Muscle Society Congress
Italfarmaco announced that additional data on givinostat for the treatment of Duchenne muscular dystrophy (DMD) are being presented at the 30th annual International Congress...
Form Bio and Cure Rare Disease Partner to Accelerate Genetic Medicine Development, Advancing CRD’s Duchenne Muscular Dystrophy Program
Form Bio, a leader in AI-powered genetic medicine development and genome engineering, announced a strategic partnership with Cure Rare Disease (CRD), a nonprofit biotechnology...