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.

DMD: Eteplirsen Results Very Encouraging at 48 Weeks

Editor's note (Oct. 3, 2012): Please read A Closer Look at the 48-Week Eteplirsen Trial Results for a more in-depth discussion of the phase 2b trial in boys with Duchenne MD.

Study of Tadalafil in BMD Revised, Reopened

Update (Nov. 9, 2012): This story has been updated to reflect that recruitment for this study ends Dec. 31, 2012.

An MDA-supported, short-term trial of the vasodilating drug tadalafil (Cialis) in teens and men with Becker muscular dystrophy (BMD) has undergone some minor restructuring and is now open to 12 additional participants.

Pages