Becker Muscular Dystrophy (BMD)

DMD/BMD Drug Screen in Zebrafish Finds Three Candidates

Three small molecules that normalize muscle structure and function and improve survival in dystrophin-deficient zebrafish have been identified by scientists at Children's Hospital Boston, Harvard Medical School, and Alaska Fisheries Science Center in Juneau.

The three chemicals, which already are approved for other medical uses, were found to increase survival and normalize muscle structure and function in the dystrophin-deficient fish without increasing  production of the muscle protein dystrophin. 

MDA Conference Brings Together Researchers, Clinicians, Industry

Moving therapeutic strategies from the laboratory to clinical trials and ultimately to the market as treatments was the theme of the MDA National Scientific Conference held March 13-16, 2011, in Las Vegas.

Some 300 people attended the conference, the first in a planned series of such MDA-sponsored meetings that will emphasize new research and current medical care. The majority of presenters and many of the audience members were current or former MDA research grantees or physicians at MDA-supported clinics.

Research Briefs: CMS, DMD, LGMD, Pompe, Stem Cells

Congenital myasthenic syndromes

A multinational team of scientists has identified mutations in the gene for glutamine-fructose-6-phosphate transaminase 1 (GFPT1) as responsible for some forms of a congenital myasthenic syndrome (CMS).

Research Briefs: CMT, CMS, DMD/BMD, FA, Pompe disease, SBMA

UCLA Researcher Receives MDA Grant to Develop DMD Drug

A new MDA translational research grant for $476,465 over three years will allow Carmen Bertoni at the University of California, Los Angeles (UCLA) to develop RTC13, an experimental compound designed to treat Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) caused by a specific type of flaw in the gene for the muscle protein dystrophin.

MDA Awards $13.5 Million in Research Grants

The Muscular Dystrophy Association has awarded 44 grants totaling $13.5 million to support research efforts aimed at advancing understanding of disease processes and uncovering new strategies for treatments and cures of muscular dystrophy and the more than 40 other diseases in the Association’s program.

The new grants were reviewed by MDA’s Scientific and Medical Advisory Committees, and approved by MDA’s Board of Directors at its December meeting.

Doctors Talk Heart to Heart

Becker MD Study Seeks Participants

A new study to determine the best "outcome measure" (measurable activity) with which to assess thigh-muscle (quadriceps) strength in men with Becker muscular dystrophy (BMD) is seeking participants.

The study, taking place at Nationwide Children's Hospital in Columbus, Ohio, is a necessary prelude to a planned trial of gene therapy involving injections of genes for the follistatin protein in people with BMD.

Experts Gather to Focus on Heart in DMD

Experts from around the world will gather Jan. 21-22, 2011, at an MDA-sponsored conference about the heart in Duchenne muscular dystrophy (DMD).

Research Briefs: DMD, BMD, MMD, SMA

Editor's note 2/7/11: A link to the Tivorsan Pharmaceuticals website has been added.

Duchenne and Becker muscular dystrophies