Peripheral Neuropathies

New MDA Grant Will Help Develop FA Drug

Development of a promising experimental medication to treat Friedreich's ataxia (FA) is proceeding, with help from a $731,534 grant MDA awarded to Repligen Corp. of Waltham, Mass., this month (December 2009).

This is the second research grant that MDA has awarded to the small biopharmaceutical company, through the Association's translational (laboratory-to-clinic) research program.

Vote Abbey for Gap Kid!

Abbey Umali needs your vote.

The vivacious 10-year-old, who is MDA’s National Goodwill Ambassador, is competing for the chance to add another title to her resume: Gap Kid.

Popular international retailer Gap has launched a nationwide search for the next faces for GapKids, its kids’ clothing line. Abbey, who has a form of Charcot-Marie-Tooth disease, is in the running and could use your help to win "fan favorite."

MDA Scientists Win Federal Stimulus Grants for Neuromuscular Disease Research

At least 15 Muscular Dystrophy Association research grantees, past and present, have won funding from the National Institutes of Health through the American Recovery and Revitalization Act (ARRA) to accelerate the search for treatments and cures for neuromuscular diseases.

DMD Idebenone Trial

This story was updated 11/9/10 with contact information for this trial and information about clinical trials in general.

Pediatric Neurologist Richard Finkel, co-director of the MDA neuromuscular disease clinic at Children's Hospital of Philadelphia, will be the lead investigator for the North American arm of a large international trial with idebenone in Duchenne muscular dystrophy (DMD).

FA Research: Bull's Eye

Scientists at the Scripps Research Institute in La Jolla, Calif., and the Repligen Corporation in Waltham, Mass., have identified the precise biochemical brake that limits production of a needed protein in Friedreich's ataxia (FA) and determined that this brake is specifically targeted by an experimental compound being developed to treat this disease. MDA is supporting Repligen to develop this drug.

FA Research: Experimental Compound Hits Molecular Target

Scientists at the Scripps Research Institute in La Jolla, Calif., and the Repligen Corporation in Waltham, Mass., have identified the precise biochemical brake that limits production of a needed protein in Friedreich's ataxia (FA)and determined that this brake is specifically targeted by an experime

Experimental Compound for FA Hits Its Molecular Target

Scientists at the Scripps Research Institute in La Jolla, Calif., and the Repligen Corporation in Waltham, Mass., have identified the precise biochemical brake that limits production of a needed protein in Friedreich's ataxia (FA) and determined that this brake is specifically targeted by an experimental compound being developed to treat this disease. MDA is supporting Repligen to develop this drug.

Skydiver with CMT

“You take one look out that door … and it’s a long way down.”

In fact it was 14,000 feet down, give or take a foot or two, for Mike Crowe and members of the U.S. Army’s elite Golden Knights precision parachuting team -– a record high for the team in tandem jumping.

Crowe, 59, of Louisville, Ky., dropped from the plane and into the sky linked to Sgt. First Class Mike Elliott, otherwise known as the tandem master.

CMT & Vitamin C

A recent trial has shown inconclusive results from various doses of vitamin C (ascorbic acid) in patients with the peripheral nerve disease type 1A Charcot-Marie-Tooth disease (CMT1A), and the jury remains out on this form of treatment, says Michael Shy, an MDA grantee at Wayne State University in Detroit.

Jury Still Out on Value of Vitamin C in CMT1A

A recent trial has shown inconclusive results from various doses of vitamin C (ascorbic acid) in patients with the peripheral nerve disease type 1A Charcot-Marie-Tooth disease (CMT1A), and the jury remains out on this form of treatment, says Michael Shy, an MDA grantee at Wayne State University in Detroit.

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