Items in this article report on clinical trials in: Duchenne muscular dystrophy and Friedreich's ataxia.
Duchenne MD gene therapy trial on track
An MDA-supported trial to test the effects of injecting a miniaturized dystrophin gene into the biceps muscles of six boys with Duchenne muscular dystrophy is moving forward without any adverse events, say investigators at Asklepios, the North Carolina biotech company that developed the gene therapy compound.
The combination of the miniaturized dystrophin gene inside an adeno-associated viral shell has been patented as Biostrophin.
Results are expected in May or June.
Idebenone, which is similar to coenzyme Q10, is being tested against a placebo for potential positive effects on heart and skeletal muscles.
The compound has been tested by Santhera and the National Institutes of Health in Friedreich’s ataxia (FA), where it has shown promise. Santhera plans to start a large-scale (phase 3) trial of idebenone in FA this summer in the United States.