The Role of Gene Therapy for Patients with Duchenne Muscular Dystrophy

CureDuchenne’s latest investment aims to address the limitations of current AAV-delivered gene therapy treatments for Duchenne muscular dystrophy (DMD).

Our recent investment in Entos Pharmaceuticals (https://entospharma.com) aims to explore the potential of their non-viral, redosable Fusogenix PLV platform to deliver full-length dystrophin protein to all muscle groups in a safe, and effective manner with lower cost compared to an adeno-associated virus (AAV) (https://entospharma.com/news-%26-media/f/entos-to-develop-dystrophin-therapy-for-dmd-with-cureduchenne).  Gene therapy is a significant t…

The Role of Gene Therapy for Patients with Duchenne Muscular Dystrophy

A panelist discusses how gene therapy using microdystrophin delivered through AAV vectors shows promise for DMD treatment, though challenges remain with delivery efficiency and determining which patients will benefit most.