Muscular Dystrophies

Experimental Drug Seems to Slow Respiratory Decline in DMD

Update (Sept. 19, 2012):This story was updated to reflect the fact that the phase 3 trial of idebenone in Duchenne MD remains open and that new sites are now participating.

NIAMS Establishes MD Research Center

A Center for Research Translation of Systemic Exon Skipping in Muscular Dystrophy has been established by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), part of the U.S. National Institutes of Health.

Jerry Mendell's Research Team Receives Award

A team headed by neurologist Jerry Mendell, a longtime MDA research grantee and director of the MDA Clinic at Nationwide Children's Hospital in Columbus, Ohio, has received the prestigious Annals of Neurology prize for an outstanding contribution to clinical neuroscience.

Man with CMD Died Doing What He Loved

Michael Wogan had been looking forward to the trip to the National Championship Air Races in Reno, Nev.

According to his younger brother James, “Michael liked to get out and travel, and he was so excited about getting on a plane.”

Michael’s older brother Billy, 26 — who, like Michael, 22, and James, 19, has congenital muscular dystrophy (CMD)— was supposed to attend the show with their dad. When he couldn’t, Michael went instead.

He promised to tell his brothers all about it.

Induced Stem Cells Require Cautious Approach

Stem cells have been much in the news lately, including for neuromuscular diseases.

PTC Remains Committed to Ataluren for Nonsense-Mutation DMD/BMD

Update (July 25, 2012): This story was updated to reflect the fact that PTC has announced its intention to begin an open-label trial of ataluren in DMD/BMD for former ataluren trial participants in Europe, Israel and Australia.

MDA Awards $13.7 Million in Research Grants

The Muscular Dystrophy Association has awarded 40 research grants totaling $13.7 million to advance the understanding of disease processes and uncover 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 recommended by MDA's Scientific and Medical Advisory Committees and approved by MDA's Board of Directors at its July 2011 meeting.

Global CMD1C/LGMD2I Registry Opens

A new registry has been launched for people with conditions caused by mutations in the gene for fukutin-related protein (FKRP). This includes people with the type 2I form of limb-girdle muscular dystrophy (LGMD) and the type 1C form of congenital muscular dystrophy (CMD) and, in rare instances, the congenital muscular dystrophies muscle-eye-brain disease and Walker-Warburg syndrome.

Families and Experts Gather at 2011 BMD Conference

Community and empowerment were the themes of the third annual Becker Muscular Dystrophy (BMD) conference held at Cedars-Sinai Medical Center in Los Angeles on August 13, 2011.

Some 150 in-person attendees, and at least that many online attenders, engaged physicians, researchers, physical therapists and other BMD experts on a variety of topics, including the latest advances in research, medical management, physical therapy and clinical trials.

Return Me to My Special Place

Artist: 

Gene transformed his love of nature into intricately detailed wood carvings of wildlife.  For many years he had a gallery, "Waterfowl by Gene," in Stockton and was active in the city's Arts Council.

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