Adapting to Change After Spinal Surgery

Tina Duong, a physical therapist at Children's National Medical Center in Washington, D.C., sees many children with neuromuscular disorders who undergo spine-straightening surgery.

Answers to the 'Critical Questions' About Eteplirsen for DMD

MDA is pushing forward on all levels to accelerate the pace at which we’re able to translate scientific discoveries into improved health outcomes for individuals living with muscle disease.

DMD, BMD: Nationwide Children's Podcast Explores Dystrophin's Interactions

In a December 2012 podcast from Nationwide Children's Hospital, cell biologist Federica Montanaro discusses her team's recent progress in understanding how various proteins interact with dystrophin and how these interactions differ in the heart versus the skeletal muscles.

MD Briefs: Gene Therapy, Exon Skipping, Stem Cells

Below is a wrap-up of recent research news about the development of therapies for Duchenne, Becker and limb-girdle muscular dystrophies.

MD Briefs: Gene Therapy, Exon Skipping, Stem Cells

Update (Jan. 23, 2013):The "Building better utrophin" section was updated to reflect the availability of a Jan. 22, 2013, press release from the University of Missouri.

Below is a wrap-up of recent research news about the development of therapies for Duchenne, Becker and limb-girdle muscular dystrophies.

UPDATED: WNT7a Injections Restore Strength in DMD Mice

Injecting a muscle-repair protein known as WNT7a into the muscles of mice lacking the dystrophin protein and showing a disease resembling Duchenne muscular dystrophy (DMD) significantly increased muscle strength and the size of muscle fibers, leading scientists to conclude that it could be developed into a treatment for DMD and the related disorder

DMD: Eteplirsen Results Still Strong at 62 Weeks

Eteplirsen, an exon-skipping drug in development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show benefit at 62 weeks with respect to the distance walked in six minutes.

What’s Next for Eteplirsen? Sarepta Discusses FDA Approval and Future Trials

In both October and December 2012, Sarepta Therapeutics announced very encouraging results from a 12-person phase 2b trial of eteplirsen, an exon-skipping compound that is a potential treatment for Duchenne muscular dystrophy.

Eteplirsen targets exon 51 of the dystrophin gene and is designed to treat DMD caused by specific dystrophin mutations.

MDA Building ‘Transitional Freedom’ Partnerships

Like many high school seniors, Drew is busy choosing a college and preparing for the big transition from life at home to life on a college campus. Unlike his peers, Drew has Duchenne muscular dystrophy.

European Agency to Consider Conditional Approval of Ataluren for DMD

New Jersey biopharmaceutical company PTC Therapeutics has announced that the European Medicines Agency (EMA) has validated a "marketing authorization application" (MAA) seeking conditional approval for ataluren, an investigational new drug for the treatment of Duchenne muscular dystrophy (DMD) or Becker muscular

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