Muscular Dystrophies

Luring Away Myostatin Can Boost Muscle Size

ACE031, a laboratory-modified protein developed by Acceleron Pharma of Cambridge, Mass., has shown promise as a therapy to increase muscle mass, based on results of a trial in healthy volunteers. The company will now test it in Duchenne muscular dystrophy (DMD).

More Good News about Exon Skipping

AVI4658, an experimental treatment for patients with Duchenne muscular dystrophy (DMD) caused by certain mutations in the gene for the muscle protein dystrophin, has shown promising results when delivered intravenously to 19 trial participants.

Laid-Off Newsman with FSHD Starts Second Career

Peter Callas Jr. remembers as if it were yesterday the day his father gave him “the F.D.R. talk.”

It was 1973, and Peter Jr., then 13 years old, had just been diagnosed with facioscapulohumeral muscular dystrophy (FSHD).

Gentamicin Shows Mixed Results in DMD

Results of the MDA-supported trial of gentamicin in Duchenne muscular dystrophy (DMD) were presented Wednesday, April 14, at the annual meeting of the American Academy of Neurology (AAN), held in Toronto.

Levels of the needed dystrophin protein increased in six out of 12 participants who received the drug for six months. No functional improvements were seen.

Progress in Exon Skipping for DMD

The multinational pharmaceutical company GlaxoSmithKline (GSK) and the Dutch biotechnology company Prosensa  announced "intriguing results" following a 12-person trial of an experimental "exon skipping" therapeutic for Duchenne muscular dystrophy (DMD).

MDA Awards Two New FSHD Grants

MDA has awarded two grants for research aimed at determining the precise molecular causes of facioscapulohumeral dystrophy (FSHD), and developing therapies for the disease.

MDA and Friends of FSH Research (FFSHR) based in Kirkland, Wash., will jointly fund a two-year, $200,000 grant to Joel Chamberlain, an assistant professor of medical genetics at the University of Washington in Seattle.

Tadalafil Trial in Becker MD

Update (Oct. 8, 2012): This story has been updated to reflect that the tadalafil trial in BMD has been slightly revised and is now open. In June 2012, it had temporarily closed to new participants. The investigators say the revised trial design was necessary because they were unable to obtain access to the MRI machine for the time periods that were necessary in the first design.

Gentamicin Trial in DMD Shows Mixed Results

An MDA-supported clinical trial of intravenous gentamicin in children and adolescents with a form of Duchenne muscular dystrophy (DMD) caused by so-called "nonsense" mutations (also called "premature stop codon" mutations) found the drug was safe and that levels of the muscle protein dystrophin increased in some, but not all, participants who received the drug for six months.

DMD Research: Potential Heart Saver

A synthetic compound that seals cellular membranes has been found to stop the progression of heart-muscle destruction in dogs with a disease closely resembling human Duchenne muscular dystrophy (DMD), MDA-supported researchers have found.

DMD/BMD Research: Ataluren Results Disappointing

The biopharmaceutical firm PTC Therapeutics announced March 3 that ataluren, its experimental drug for certain forms of Duchenne (DMD) and Becker (BMD) muscular dystrophy, although safe and well tolerated, failed to meet its primary end point within the 48-week duration of the phase 2b trial. That end point was an improvement in how far boys with DMD or BMD could walk in six minutes.

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