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

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

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.

Young Jerry

Artist: 
Tommy Roberts

Tommy began creating art in childhood.  He has a bachelor’s degree from the Maryland Institute, College of Art in Baltimore.  He is well known in the Baltimore and Washington areas for his exceptional work.  Tommy has the ability to capture his subject’s emotion and character as seen in this portrait of Jerry Lewis who Tommy watched in movies in the 1960s and 1970s.  He has three other pieces in the MDA Art Collection.

Study of Tadalafil in BMD Revised, Reopened

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.

Lack of availability of the MRI (magnetic resonance imaging) device during specific periods of time was the reason for redesigning the trial, according to principal investigator Ronald Victor.

BMD Conference Videos Cover Health Care, Research and Daily Living

Videos of the entire MDA-sponsored Becker Muscular Dystrophy Conference, held in Chicago on Aug. 11, 2012, are now archived on the Conference video page.

MDA Commits $10.7 Million to Neuromuscular Disease Research

The Muscular Dystrophy Association has awarded 33 new grants totaling $10,684,481 to fund research projects focused on uncovering the causes of, and developing therapies for, neuromuscular disease.

MDA's Board of Directors reviewed and approved the new grants based on recommendations from the Association's Scientific and Medical Advisory Committees, and the grants took effect Aug. 1.

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