Findings from a study of 516 Italian boys show a significant improvement over the last six decades in survival time in Duchenne muscular dystrophy (DMD).

The multinational pharmaceutical company GlaxoSmithKline (GSK) has announced promising results for its phase 1 trial of the exon-skipping drug drisapersen in boys with Duchenne muscular dystrophy (DMD) who are no longer walking.

The phase 1 trial was designed to test safety, tolerability and pharmacokinetics (what the body does to the drug) of drisapersen, not to test drug efficacy.

Compounds targeting exon 52 and exon 55 of the dystrophin gene as a way to treat some forms of Duchenne muscular dystrophy (DMD) will be moved into clinical trials as soon as possible, says Dutch biotechnology company Prosensa. The experimental compounds are known as PRO052 and PRO055.

A multicenter study of the use of magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) to measure the progression of Duchenne muscular dystrophy (DMD) is open at sites in Florida, Oregon and Pennsylvania for boys with DMD who meet study criteria.

The biopharmaceutical company Sarepta Therapeutics announced today that its experimental exon-skipping compound, eteplirsen, resulted in an increase in dystrophin and  "significant clinical benefit" on the six-minute walk test in a phase 2b trial in boys with Duchenne muscular dystrophy (DMD) caused by specific mutations.

The experimental exon-skipping drug eteplirsen, in development by Sarepta Therapeutics to treat approximately 13 percent of boys with Duchenne muscular dystrophy (DMD), has been found to have significant functional and biochemical benefits in a phase 2b trial.