Biopharmaceutical company Sarepta Therapeutics has announced it will expand the focus of its exon-skipping program for Duchenne muscular dystrophy (DMD) by developing compounds that target exons 45, 50 and 53 of the dystrophin gene, in addition to continuing to develop eteplirsen, which targets ex

The drug tadalafil (Cialis, Adcirca), which dilates blood vessels and is approved to treat erectile dysfunction and pulmonary hypertension, has been found to improve blood flow to exercising forearm muscles in people with Becker muscular dystrophy (BMD). Enhancing blood flow may reduce damage related to muscle contraction, although this has not yet been established.

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).

Scientists funded in part by MDA have found the cause of type 2facioscapulohumeral muscular dystrophy (FSHD), a discovery that increases scientific understanding of both forms of FSHD, will improve diagnosis of the disease and could lead to treatment advances.

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.

By disrupting the fukutin gene at different time points in mice embryo, researchers have been able to develop research models of two types of human muscle disease: Fukuyama congenital muscular dystrophy (Fukuyama CMD)  and type 2M limb-girdle muscular dystrophy (LGMD2M).

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.