Becker Muscular Dystrophy (BMD)

Muscle Physiology — Masahiro Iwamoto, Ph.D., D.D.S.

Masahiro Iwamoto, research scientist at the Children’s Hospital of Philadelphia and associate professor of pediatric orthopedics at the University of Pennsylvania School of Medicine, was awarded an MDA research grant totaling $405,000 over a period of three years to study new ways to reduce muscle degeneration.

DMD, BMD — Joseph Beavo, Ph.D.

Joseph Beavo, professor of pharmacology at the University of Washington in Seattle was awarded an MDA research grant totaling $412,500 over a period of three years to study how the drug sildenafil acts to improve the function of heart muscle in animal models of Duchenne muscular dystrophy (DMD).

BMD, DMD — Linda Baum, M.D., Ph.D.

Linda Baum, professor and vice chair of pathology and laboratory medicine at the Geffen School of Medicine at the University of California, Los Angeles, was awarded an MDA research grant totaling $405,000 over a period of three years to study molecules on the muscle surface that regulate important aspects of cellular communication and survival.

DMD: Dystrophin-Deficient Dogs Benefit from Gene Therapy

For the first time, gene therapy using a highly miniaturized dystrophin gene resulted in significant improvement in muscle structure and function in dogs with a disorder mimicking human Duchenne muscular dystrophy.

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

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.

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

Update (Dec. 6, 2012): This story has been updated with additional information about next steps for ataluren.

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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