Spinal Muscular Atrophy (SMA)

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

$1.4 Million MDA Grant Will Help Develop SMA Drug

MDA has awarded a $1.4 million grant to the biopharmaceutical company Repligen Corp. to help advance the company’s experimental drug for spinal muscular atrophy (SMA) to phase 1 human clinical trials.

The drug, RG3039, has demonstrated potential throughout its early development, and is Repligen's lead therapeutic candidate for treatment of SMA.

Research Briefs: DMD, BMD, CMD, SMA

Duchenne and Becker muscular dystrophies

Survey Examines Reproductive Choices in Families with SMA

Parents of children with spinal muscular atrophy (SMA), a genetic disease of the nervous system that shows variable severity, make different decisions about whether or not to have more children.

Katelyn Hanson, a genetic counseling student at Indiana University School of Medicine, and Virginia Thurston, clinical associate professor of medical and molecular genetics at the same institution, are conducting a study to find out more about the factors that influence decisions made by parents of SMA-affected children.

NIH Convenes Meeting on SMA Treatment Development

In a sign of the significant progress being made in spinal muscular atrophy (SMA) research, the National Institutes of Health (NIH) convened a meeting in October that examined the status of laboratory research, clinical trials and strategies for drug development in this disease, with an eye toward speeding the process.

Power Soccer Champ with SMA Excels on Multiple Fronts

Ben Carpenter, a young man with many talents.

Don’t be surprised if, in a few years, you hear about some fascinating new amusement park rides. And don’t be surprised if a young man named Ben Carpenter proves to be the genius behind the design of those rides.

Deshae Lott Scholarships Awarded

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.”

Research Briefs: ALS, CMD, FA and SMA

Amyotrophic lateral sclerosis (ALS)

US Team Wins PowerHockey Cup

It didn’t look good for the Minnesota Saints.

Playing against the tough Michigan Mustangs for the top prize in power wheelchair hockey, the Saints went up 4-2, only to see the score tied 4-4 with just five minutes to go.

That’s when forward Chad Wilson, 21, of Chanhassen, Minn., went into overdrive. Wilson, who has Becker muscular dystrophy (BMD), took less than two minutes to score two more goals for the Saints.

Research Briefs: ALS, BMD, DMD, MMD, SMA, Muscle Regeneration

Amytrophic lateral sclerosis (ALS)

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