News & Media
April 19, 2019
Long-Term Follow Up of Patients Receiving Novel Gene Therapy for SMA Type I
Researchers recently published the long-term outcomes of patients who received the investigational drug AVXS-101 – an adeno-associated virus serotype 9 mediated gene replacement therapy.
November 16, 2018
Big Gains in FSHD Research: A Newly Published Model of FSHD and a Potential Gene Therapy to Improve Functional Outcomes
The model aims to provide the basis for many future studies to bring therapeutic options to patients with FSHD.
July 24, 2018
Preclinical Gene Therapy Study Shows Muscle Restoration in Charcot-Marie-Tooth Disease Model
Tests of NT-3 gene therapy on mouse models suggest potential to treat a host of muscle-wasting conditions.
April 2, 2018
Can Gene Therapy Treat Dominantly Inherited Disorders?
Recent applications of adeno-associated virus (AAV) mediated gene therapy have focused mainly on correcting recessively inherited diseases. But what about dominantly inherited disorders? It looks like AAV could be a delivery mechanism for treating those genetic disorders, too.
September 20, 2016
Nationwide Children’s Hospital Leads Effort for Gene Therapy Treatment for Duchenne Muscular Dystrophy
Sarepta Therapeutics announced yesterday that the FDA has granted accelerated approval for eteplirsen for the treatment of Duchenne muscular dystrophy...