Peripheral Neuropathies

$13.6 Million in New MDA Grants Promote Understanding, Treatment of Neuromuscular Diseases

The Muscular Dystrophy Association has awarded 44 new grants totaling $13.6 million to advance the understanding and treatment of neuromuscular diseases. The new grants, most of which took effect Feb. 1, encompass a range of diseases covered by MDA’s research program, and they support innovative approaches to basic research and new drug development.

In addition to addressing 16 specific neuromuscular diseases under MDA’s umbrella, the grants also fund research into muscular dystrophy in general, and research into muscle physiology related to neuromuscular disease.

Spice for CMT1B? Curcumin Shows Benefit in Research Mice

Mice with a disorder resembling the type 1B form of Charcot-Marie-Tooth disease (CMT) benefited from treatment with either of two forms of oral curcumin, a component of the spice turmeric, according to researchers supported in part by MDA.

Friedreich’s Ataxia: Drug Enters Phase 2b Clinical Trial

Edison Pharmaceuticals has launched a phase 2b clinical trial of its experimental drug EPI-743 in adults with Friedreich's ataxia (FA) to assess whether the drug has positive effects on visual function, neurological and neuromuscular function, and disease-associated biomarkers.

Experimental Drug Being Tested in Mitochondrial and Metabolic Disorders, FA

Paralympian with Mitochondrial Myopathy Swims in London

Update (Oct. 16, 2012): Joe Wise placed fifth in the 400-meter freestyle at the London Paralympic Games. Watch an interview with Joe to learn more about his experiences at the Paralympics.

MDA Commits $10.7 Million to Neuromuscular Disease Research

The Muscular Dystrophy Association has awarded 33 new grants totaling $10,684,481 to fund research projects focused on uncovering the causes of, and developing therapies for, neuromuscular disease.

MDA's Board of Directors reviewed and approved the new grants based on recommendations from the Association's Scientific and Medical Advisory Committees, and the grants took effect Aug. 1.

New Gene Therapy Approach for FA, Other Diseases

Human cells treated with engineered transcription activation-like effector (TALE) proteins produced two to three times more frataxin protein than did control cells, a team of researchers has reported.

Researchers Studying Children's Use of Mobility Devices

If you're the parent of a young child who uses a power mobility device, researchers at the University of British Columbia (UBC) and Sunny Hill Health Centre for Children — both in western Canada — are interested in hearing from you. The study is open to U.S. residents.

FA: Immune System Drug Strengthens Mice

Treatment with an engineered version of the naturally produced interferon gamma protein enhanced the ability to move, and improved balance and coordination in mice with a disease resembling Friedreich's ataxia (FA), a team of researchers has reported. In addition, treatment with interferon gamma prevented degeneration of sensory neurons (nerve cells) in the mice.

Decision Making About PGD Is Complex, Study Finds

Decision making about preimplantation genetic diagnosis (PGD) is a complex, multiphase process for couples, a new study has found. Understanding it, the investigators say, may be helpful to prospective parents who know they're at risk for transmitting a genetic disorder, and to the professionals who advise them.

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