News & Media


May 28, 2019

Preventing the Development of Muscular Dystrophy Through Surrogate Gene Therapy

Researchers identify a key regulatory protein implicated in Galgt2 overexpression and begin to elucidate its protective mechanism against muscular dystrophy.


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.

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