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

Lee Breidenbach

2012
Full name: 
Mr. Lee Breidenbach

Untitled - Lee Breidenbach

Artist: 
Lee Breidenbach
Disease: 
Duchenne Muscular Dystrophy (DMD)
Medium: 
Watercolor

Wilson Long

2012
Full name: 
Mr. Wilson Long

Looking a Head

Artist: 
Wilson Long
Disease: 
Duchenne Muscular Dystrophy (DMD)
Medium: 
Collage

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.

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

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.

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