overview

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: GSK Returns Drisapersen Development Rights to Prosensa

The rights to development of the experimental Duchenne muscular dystrophy (DMD) drisapersen have now been returned to the drug's original developer, Dutch biotechnology company Prosensa. The rights were formerly jointly held by Prosensa and multinational pharmaceutical company GlaxoSmithKline (GSK).

College Course on DMD Available Online

A course on Duchenne muscular dystrophy (DMD), geared toward graduate and upper-division undergraduate students and held at Ohio State University College of Medicine and Nationwide Children's Hospital in Columbus, is open to the public, without charge, via real-time streaming and podcasts.

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.

Survey for People with IBM Reports Early Findings

Investigators conducting an online survey launched in January 2013 to gather anonymous information from people with inclusion-body myositis (IBM) are now reporting preliminary results and are asking the original respondents to complete a short supplementary survey.

CNM/MTM Registry, World Map Seek Participants

A patient registry and world map of people with centronuclear myopathies (CNM), including myotubular myopathy (MTM), are being developed and are seeking participation from people with these disorders or their family members.

Becker MD Conference Can Be Attended Online Aug. 24

MDA and John Hopkins Medicine will present the 2013 national Becker Muscular Dystrophy Conference Saturday, August 24, in Baltimore. Free to all participants with BMD and their family and friends, the conference can be attended in person or online.

MDA Funds Study of Prednisone in Very Young Boys with DMD

Pediatric neurologist Anne Connolly has received an MDA grant to study the effects of prednisone on Duchenne muscular dystrophy in very young children.

DMD: Sarepta to Submit New Drug Application to FDA for Eteplirsen in 2014

 In a July 24, 2013, press release and conference call, biotechnology company Sarepta Therapeutics provided an update on the regulatory pathway for eteplirsen, the company’s experimental drug for Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin

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