Duchenne Muscular Dystrophy (DMD)

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).

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.

Alex Vargas

2012
Full name: 
Alex Vargas
Artist: 
Alex Vargas
Disease: 
Duchenne Muscular Dystrophy (DMD)
Medium: 
Acrylic

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.

DMD: Reformulated Utrophin Upregulator Looks Good in Healthy Volunteers

Update (Nov. 7, 2012): In a Nov. 7, 2012, press release, Summit PLC announced that twice-daily oral dosing of SMT C1100 in healthy volunteers appears safe and well-tolerated, and results in blood levels of the drug that would be expected to increase utrophin production.

DMD Imaging Study Open at Three Sites

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.

A Closer Look at the 48-Week Eteplirsen Trial Results

Editor's note (Oct. 26, 2012): This story has been modified to note that it's possible for the U.S. Food and Drug Administration to give conditional approval to a promising rare-disease drug prior to a larger confirmatory trial being performed.

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