Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

With the first GMP batch of Trogenix's rAAV vector manufactured at ... set to form a partnership with Axovia Therapeutics to progress the development and manufacturing of an AAV9-based gene ...

CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new DMD gene therapy.

SRF published this paper to document and share the critical progress made in advancing SYNGAP1 therapeutic development and to share our roadmap for other patient advocacy groups (PAGs) focused on ...