Becker Muscular Dystrophy (BMD)

Drug Development for DMD: Fall 2014 Update-Part 2

Several experimental drugs are i development to treat Duchenne muscular dystrophy (DMD), a genetic disorder that results in a lack of the dystrophin protein in cardiac and skeletal muscle cells.

BMD: Follistatin Gene Transfer Results Encouraging

Results from a trial involving injection of genes for the follistatin protein into the thigh muscles of both legs in six men with Becker muscular dystrophy (BMD) show the experimental treatment appears safe and and was associated with improvement in walking ability (distance walked in six minutes) in four of the six trial participants.

DMD: Encouraging Results for SMT C1100

Tadalafil, Sildenafil Increase Blood Flow to DMD-Affected Muscles

MDA Thrilled FDA Will Consider Accelerated Approval for New Muscle Disease Drug Aimed at DMD

New Forms of Genetic Testing Improve Diagnosis, Raise Questions

“Knowing, if not all, is almost all,” said Matthew Harms, a neurologist and neurophysiologist from Washington University in St. Louis, in his presentation on genetic testing for neuromuscular disorders at the 2014 MDA Clinical Conference, held in Chicago March 16-19.

MD-CARE Act Congressional Briefing Accelerates Reauthorization Momentum

"The energy in the room was high, and the mood was optimistic," said Annie Kennedy, MDA's senior vice president of advocacy, speaking about the Congressional briefing on reauthorization of the MD-CARE Act that she attended and helped to organize. The briefing was co-hosted by MDA and other muscular dystrophy organizations in Washington, D.C., on Feb.

DMD, BMD: Ataluren Did Not Receive Early Approval in Europe

DMD/BMD: Stop Codon Read-Through Drug Trial Still Recruiting Participants

Study Seeks Parents, Guardians of Young Children with Neuromuscular Disease

The National Institute of Nursing Research, part of the National Institutes of Health (NIH) in Bethesda, Md., is seeking parents or guardians of children from birth through age 5 with a neuromuscular disease for a study to develop a questionnaire about motor function in young children.

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