Editor's note 2/7/11: This article was updated to reflect that Pallavi Anand is the primary coordinator for the Missouri site.

Finding out whether or not an experimental treatment helps individuals with Duchenne muscular dystrophy (DMD) requires that researchers know the usual course of the disease and have measurements that will show if a treatment is changing the disease course.

A large-scale, phase 3 trial of idebenone (Catena) in Duchenne muscular dystrophy (DMD) is now open at one U.S. site and several sites in Europe, under the auspices of Santhera Pharmaceuticals.

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.

Luring away myostatin, and possibly other proteins that inhibit muscle growth, using a gene for a "decoy receptor" increased muscle size and strength in mice with a muscle disease resembling human Duchenne muscular dystrophy (DMD), a new study has found.

The National Institutes of Health (NIH) has awarded $7 million to Nationwide Children's Hospital in Columbus, Ohio, and designated the institution a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center (Wellstone MDCRC).

MDA has awarded 38 new research grants totaling more than $14 million and covering more than a dozen neuromuscular diseases. 

MDA's Board of Directors met in Los Angeles July 16, where it reviewed and approved the new grants based on recommendations from the MDA Scientific and Medical Advisory Committees. Grants were scored and recommended for approval based on the capabilities of the applicant, the scientific merit of the project, and the proposal's relevance to developing treatments for the disease. The effective start date for all grants was July 1, 2010.

Update (Nov. 5, 2012): Results announced in November 2012 showed the drug, now called drisapersen, reached blood levels approximately proportional to the injected dose at two of the three dosage levels tested and was not associated with serious adverse events. See Drisapersen Appears Safe in Non-Walking Boys With DMD.

The biopharmaceutical company AVI BioPharma has announced additional encouraging results from its clinical trial of AVI4658, an experimental treatment for Duchenne muscular dystrophy (DMD).

The new results show that, at higher doses, AVI4658 can result in substantial production of the needed dystrophin protein in muscle fibers.

The company has not yet released results of any tests of muscle function in the 19 children in this trial.