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Hope Through Research

MDA is the world leader in sponsoring research seeking the causes of, and effective treatments for, neuromuscular diseases.

Almost every treatment currently in development for neuromuscular diseases is based on research funded by the Muscular Dystrophy Association.

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Research News

[title]
FA: RG2833 is Well-Tolerated and Increases Frataxin Gene Activity
May 17, 2013
Interim results from a phase 1 clinical trial of RG2833 in people with Friedreich’s ataxia (FA) show that the experimental drug is well-tolerated,... Read More
[title]
Newborn Screening Recommended for Pompe Disease
May 17, 2013
The Discretionary Advisory Committee on Heritable Disorders in Newborns and Children (DACHDNC) today voted to add Pompe disease (acid maltase... Read More
[title]
Trial Tests 'Master Regulator' in ALS
May 16, 2013
A phase 2 clinical trial to test the experimental drug GM604 in people with amyotrophic lateral sclerosis (ALS) has opened at two trial sites in the... Read More
[title]
Melatonin is Neuroprotective in ALS Mice
May 10, 2013
Melatonin, a naturally occurring hormone produced by the pineal gland, is best known for its role in regulating sleep. Now, results from a study... Read More
[title]
DMD, BMD: Phase 3 Trial Opens for Stop Codon Read-Through Drug
May 08, 2013
A large-scale, multinational phase 3 trial of the experimental drug ataluren has opened its first trial site, in Cincinnati, Ohio.The trial is... Read More
[title]
SMA: Trial Tests Antisense Therapy in Infants
May 07, 2013
A phase 2 clinical trial to test multiple doses of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA) has opened at four... Read More
[title]
MTM Phone-Based 'Event' Study Now Open
May 03, 2013
The Myotubular Myopathy Event Study, a telephone-based survey, will gather information about MTM-associated events, such as emergency room visits,... Read More
[title]
UPDATE: ACE-031 Clinical Trials in Duchenne MD
May 02, 2013
Biopharmaceutical companies Acceleron Pharma and Shire announced April 21, 2011, that MDA-supported clinical trials of ACE-031 for Duchenne muscular... Read More
[title]
MDA Scientific Conference Blog: We Need Each Other
April 25, 2013
[title]
MDA Scientific Conference Blog: Playing in the Same Sandbox
April 25, 2013
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Research

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    • Volume 1, Issue 1, October 2011
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Muscular Dystrophy Association — USA
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