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

DMD Progression Studied in Very Young and Nonwalkers

Parents’ Perceptions Sought of DMD/BMD Ataluren Trials

If your child participated in a phase 2 clinical trial of ataluren (PTC124) for Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) conducted by PTC Therapeutics, you're invited to take part in a National Institutes of Health (NIH) study about the experience.

Large-Scale Trial of Idebenone in DMD is Now Open

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.

Longer Chromosome Tips Mean Better Muscle Repair in DMD mice

Dystrophin-deficient mice (known as mdx mice) have been used in experiments as a model of human Duchenne muscular dystrophy (DMD) for decades. However, scientists have long noted that mdx mice, even though they develop a disease that mimics some aspects of human DMD, fare much better than human DMD patients. Mdx mouse muscles don't deteriorate as severely, and their ability to move and maintain heart function is far better than that of humans with the disease.

Research Briefs: DMD, BMD, CMD, SMA

Duchenne and Becker muscular dystrophies

Viagra May be Heart Helper in DMD

Results from a recent study have shown that treatment with the drug sildenafil (brand name Viagra) conferred both long-term protection against cardiac (heart) dysfunction in younger mice, and rapid reversal of heart damage in aged mice with a disease resembling Duchenne muscular dystrophy (DMD).

Research Briefs: DMD

Anti-myostatin drug trial shows good preliminary results

Immune Response Must Be Considered in DMD Gene Therapy

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.

NIH Continues Funding for MD Research

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.

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