Muscular Dystrophies

Anti-myostatin drug trial shows good preliminary results

The experimental drug AVI4658, in development by AVI BioPharma to treat Duchenne muscular dystrophy (DMD) caused by specific genetic mutations, was well tolerated and resulted in increased production of the needed dystrophin protein. Measured aspects of cardiac, pulmonary and skeletal muscle function remained stable.

A low-dose regimen of ataluren (formerly called PTC124), an experimental drug developed by PTC Therapeutics to treat  Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) caused by a certain type of genetic mutation, is superior to a high-dose regimen or a placebo.

Editor's note: This story was updated April 27, 2012.

Immune-system rejection of newly synthesized dystrophin protein occurred in at least some of the boys with Duchenne muscular dystrophy (DMD) who participated in a safety trial of dystrophin gene therapy.

The finding was a partial surprise to researchers and demonstrates the value of small, phase 1 clinical trials.

This story was updated Oct. 6, 2010.

Researchers, clinicians, pharmaceutical industry executives, and representatives from advocacy groups, including MDA, met in Washington Sept. 27-28, 2010, to discuss moving forward with antisense-based therapies for neuromuscular disease.

The U.S. National Institutes of Health (NIH) announced Sept. 29, 2010, that it will allocate more than $4.5 million for the first year of a five-year commitment to explore new treatment strategies for various forms of muscular dystrophy.

Support will go to three U.S. institutions: Nationwide Children's Hospital in Columbus, Ohio; the University of Pennsylvania in Philadelphia; and the University of Iowa in Iowa City.

Duchenne muscular dystrophy

Three accomplished scholars determinedly pursuing their educations while living with neuromuscular disease are the first winners of the CMMS Deshae Lott Ministries outreach program scholarships.

“We were so impressed,” with the winning candidates, said Deshae Lott, 39, a teacher and minster from Bossier City, La., who founded the nonprofit that bears her name. “These three show great strength of mind and character. Each of them strives to maximize within their limitations.”

A new study reported by the Centers for Disease Control and Prevention (CDC) shows that survival time has significantly increased for certain categories of people with muscular dystrophy (MD) but that race and cardiac status have a large impact on survival.