Muscular Dystrophies

DMD: Sarepta Expands Exon-Skipping Program

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

Tadalafil Increases Muscle Blood Flow in Becker MD

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.

WNT7a Injections Restore Strength in DMD Mice

Update (Dec. 20, 2012):This story has been updated to reflect the availability of a 12-minute podcast with researcher Michael Rudnicki, in which Rudnicki discusses the advantages and disadvantages of developing protein-based therapies (such as WNT7a) versus cell-based therapies to treat muscle disease.

Study Shows Increased Life Span in DMD in Recent Decades

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 Find Cause of Type 2 FSHD

Update (Nov. 14, 2012): This story has been updated to reflect information about genetic testing.

Drisapersen Appears Safe in Non-Walking Boys with 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.

DMD: Prosensa, GSK Expand Exon-Skipping Program

Update (Feb. 1, 2013): Dutch biopharmaceutical company Prosensa announced Jan. 29 that it has received orphan drug status in the United States and the European Union for compounds in development for the treatment of Duchenne muscular dystrophy.

Anti-Cancer Drugs May Help Build Muscle

Experimental anti-cancer drugs that block IAP (inhibitor of apoptosis) genes and kill cancer cells have unexpectedly been found to induce the growth and repair of muscle tissue, say scientists supported in part by MDA.

Ventricular Assist Device Implanted in Man with Duchenne MD

A left ventricular assist device (LVAD), which helps the heart pump blood throughout the body, was implanted in the chest of a young man with Duchenne muscular dystrophy (DMD) at Cincinnati Children's Hospital Medical Center in September. The patient is 29-year-old Jason Williams, of Peebles, Ohio.

Nationwide Children’s Podcast Explores the Congenital Muscular Dystrophies

Editor's note (Oct. 19, 2012): This story was updated to reflect the fact that Kevin Flanigan co-directs the MDA Clinic at Nationwide Children's Hospital in Columbus, Ohio.

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