Duchenne Muscular Dystrophy (DMD)

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.

MDA Muscle Symposium: Newborn Screening in DMD

Update (Feb. 8, 2013): Longtime MDA grantee Jerry Mendell, who directs the Center for Gene therapy at Nationwide Children's Hospital in Columbus, Ohio, published Report of MDA Muscle Disease Symposium on Newborn Screening for Duchenne Muscular Dystrophy in Muscle & Nerve, Feb. 8, 2013.

DMD Briefs: Stopping Inflammation on Earth and in Space

Inflammation— the immune system's first line of defense in tissue that's been damaged by injury or infection — is a good example of a process that's a good thing up to a point and under certain circumstances and a bad thing in excess or under the wrong circumstances.

Sarepta Issues ‘Open Letter’ Explaining Development of Eteplirsen for DMD

In July 2012, Sarepta Therapeutics announced encouraging interim results for its phase 2b trial of eteplirsen, an experimental exon-skipping drug in development to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene.

MDA Applauds Congress for Including Two Neuromuscular Diseases in Pediatric Research Bill

In July, days before the U.S. House of Representatives and Senate went into recess, bipartisan legislation was introduced in both houses to strengthen research into pediatric diseases.

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