Unconventional Pianist with CMT Hopes to Raise a Million

Amble playing a "techno music" piece that he composed when he was 9.

Research Briefs: CMT, CMS, DMD/BMD, FA, Pompe disease, SBMA

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.

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.

Narrowing Down CMT Subtypes

 

Editor's note:This story was updated Feb. 4, 2011, to reflect the availability of a paper and editorial on this subject in Annals of Neurology.

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.

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.

MDA Mailbag Spring 2009

Letters to Quest: Camp is so important ** Moms on Wheels ** All camps not the same ** No guarantees (life expectancy) ** FMLA and sibling caregivers ** Pay for family caregivers.

Speedier Social Security Benefits for Four Muscle Diseases

Annie Kennedy

Applying for Social Security benefits can be tedious and frustrating, often resulting in the first application being denied and initiating a long appeal process.

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