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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.

High-Calorie Diet May Help in ALS

A study supported in part by MDA has found that a high-calorie, high-carbohydrate diet is safe and well-tolerated in late-stage amyotrophic lateral sclerosis (ALS), that it leads to moderate weight gain, and that it may slow disease progression.

SMA: Interim Results Look Promising for SMN-Raising Drug

Interim results of a phase 2 clinical trial to test multiple doses of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA) suggest that the drug is well-tolerated and may prolong ventilator-free survival.

DMD: Prosensa to Present Webcasts on Drug Development

Dutch biopharmaceutical company Prosensa, developer of the experimental drug drisapersen and other experimental compounds for Duchenne muscular dystrophy (DMD), will update the investor community via a Thursday, Jan. 16, 2014, presentation at the J.P. Morgan Healthcare Conference in San Francisco. The presentation will be webcast at 10 a.m. Pacific time/1 p.m. Eastern time.

On Tuesday, Jan. 21, at 8 a.m. Eastern time, Prosensa will conduct a separate webinar for DMD patients and families.

DMD: Eteplirsen-Treated Boys Show Continued Stability at 120 Weeks

Boys with Duchenne muscular dystrophy (DMD) participating in a phase 2b extension trial of the experimental exon-skipping drug eteplirsen showed continued stabilization of walking distance at 120 weeks. Sarepta Therapeutics, developer of eteplirsen, announced the findings in a Jan. 15, 2014, press release.

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.

International ALS Symposium Stresses Progress

"We are undoubtedly slowing down the MND [motor neuron disease] supertanker, and can start to see how we might turn it around for the first tim," said Martin Turner, senior clinician scientist at the University of Oxford (United Kingdom), on research being reported on at the 24th International Symposium on ALS/MND.

Blood-Filtering Procedure Removes Antibodies That Impede Gene Transfer

A research group supported in part by MDA has found that a blood-filtering strategy known as plasmapheresis looks promising as a way to help overcome one type of unwanted immune response to gene transfer therapy. Gene transfer therapy is the addition of therapeutic genes to treat disease, and it is often administered via transport vehicles made from viruses.

Louis Chicoine at Nationwide Children's Hospital and Ohio State University in Columbus, Ohio, and colleagues, reported the findings online Oct. 23, 2013, in the journal Molecular Therapy.

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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