Peripheral Neuropathies

MDA Awards $13.5 Million in Research Grants

The Muscular Dystrophy Association has awarded 44 grants totaling $13.5 million to support research efforts aimed at advancing understanding of disease processes and uncovering new strategies for treatments and cures of muscular dystrophy and the more than 40 other diseases in the Association’s program.

The new grants were reviewed by MDA’s Scientific and Medical Advisory Committees, and approved by MDA’s Board of Directors at its December meeting.

Biobank Collecting Blood Samples for Neuromuscular Disease Research

People with genetic neuromuscular diseases who want to “do something for science” now have a way to do so, although they’re unlikely to ever know the results of their good deed.

Young Author with CMT Pens Tale of High-Flying Goose

Ruthie B. Goose is by no means your average goose.

Ruthie has very high expectations of herself — specifically to fly higher than any other bird has flown before.

Ruthie’s resolution and her efforts to fly to 37,901 feet are the subject of a book written by a 10-year-old girl and her father, both of whom have Charcot-Marie-Tooth disease (CMT).

Two-Drug Combo Makes a Difference in FA

Results from a clinical trial conducted in Spain involving 20 individuals with Friedreich's ataxia (FA) show that a combination therapy using two drugs, idebenone and deferiprone (DFP), demonstrated a "stabilizing effect" in neurological function and reduced abnormal heart-muscle enlargement in participants who received the two-drug combo for a period of 11 months.

Research Briefs: DMD, FA, DM, PM, IBM, MG, LEMS

Duchenne muscular dystrophy

Research Briefs: ALS, CMD, FA and SMA

Amyotrophic lateral sclerosis (ALS)

MDA Awards More Than $14 Million in Research Grants

MDA has awarded 38 new research grants totaling more than $14 million and covering more than a dozen neuromuscular diseases. 

MDA's Board of Directors met in Los Angeles July 16, where it reviewed and approved the new grants based on recommendations from the MDA Scientific and Medical Advisory Committees. Grants were scored and recommended for approval based on the capabilities of the applicant, the scientific merit of the project, and the proposal's relevance to developing treatments for the disease. The effective start date for all grants was July 1, 2010.

FA Research: Idebenone Strikes Out Again

Idebenone, a substance believed to act as an antioxidant and to aid in energy production in cellular structures called mitochondria, has failed to benefit people with Friedreich's ataxia (FA) in a phase 3 trial conducted at several European centers. The drug is similar to coenzyme Q10, a naturally occurring molecule.

New Tools for Trials in Children with CMT

Two scales that can be used to assess disease progression and any response that may occur to a treatment have been developed for children with Charcot-Marie-Tooth (CMT) disease.

One, called the CMT Pediatric Scale, measures physical functioning. The second, called the Pediatric CMT Quality of Life Instrument, measures how the disease affects children's life experience. The two scales eventually will be merged, the investigators say.

Curcumin Helps Mice With CMT-Like Disease

Mice with a genetic mutation in the myelin protein zero (MPZ) gene, which develop a disease resembling human type 1B Charcot-Marie-Tooth disease (CMT1B), benefited from treatment with curcumin and curcumin derivatives, researchers announced April 15, at the 2010 meeting of the American Academy of Neurology (AAN), held in Toronto.

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