Gene editing with adeno-associated virus vector offers hope for hereditary deafness
An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent ...
GEN - Genetic Engineering and Biotechnology News11d
DMD Patient Dies After Treatment with Sarepta Gene Therapy
The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...
The American Journal of Managed Care11d
Shaping Gene Therapy Approaches in Neuromuscular Disease
Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...
Technology Networks10d
Gene Therapy Offers Hope for Dravet Syndrome Treatment
Scientists developed a promising gene replacement therapy for Dravet syndrome in mice. The therapy alleviated symptoms ...
EurekAlert!11d
Breakthrough Dravet syndrome gene therapy in mice brings new hope to families
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
Sarepta Therapeutics Reports Patient Death Post Elevidys Gene Therapy Treatment, Stock Plunges
Sarepta Therapeutics, Inc. (NASDAQ:SRPT) stock is sliding. The company shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne ...
Fierce Pharma11d
Sarepta reports one death after patient received DMD gene therapy Elevidys
One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech ...
Benzinga.com12d
Sarepta Therapeutics Reports Patient Death Post Elevidys Gene Therapy Treatment, Stock Plunges
Elevidys is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy ...