MDA News and Updates

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ABC Television Network Is New Home of MDA Show of Strength Telethon
June 17, 2013
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ALS Briefs: New Findings About Three ALS-Associated Genes
June 13, 2013
Recent research in amyotrophic lateral sclerosis (ALS) includes findings involving the CRMP4, FUS and TDP43 genes.
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DMD: 'Permanent' Gene Repair Strategy Looks Good in Lab
June 11, 2013
Permanent repair of a faulty gene has long been a goal of researchers working to develop gene-based therapies. But many current gene modification strategies that have entered clinical trials have been based on temporary forms of gene correction — treatments that will need to be given frequently throughout a person's life.
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Genzyme and MDA Provide Pompe Families with Free Testing
June 10, 2013
Pompe disease (acid maltase deficiency, or GAA deficiency) shares symptoms with several other disorders and, in the absence of specific testing, can sometimes be confused with these other conditions.
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MD Briefs: Registries Are Still Open
June 07, 2013
FSHD, MMD: Registry remains open
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ALS Briefs: Disease Modifier, Growth Factors, Antioxidant Genes
June 06, 2013
Identification of the biological mechanisms that underlie amyotrophic lateral sclerosis (ALS) may provide scientists with important clues about the disease that may be used to identify
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New Muscular Dystrophy Drugs Offer Hope
June 04, 2013
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Study Suggests Sleep-Disordered Breathing Common in FA
June 04, 2013
Results from a study conducted in Australia show that a nighttime respiratory problem called obstructive sleep apnea occurs more frequently in people with Friedreich's ataxia (FA) than in the general population.
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Exome Sequencing Links New Genes to Sporadic ALS
May 31, 2013
A team of researchers in the U.S.
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MTM: MDA Funds Development of Myotubularin-Based Treatment
May 29, 2013
The Muscular Dystrophy Association has awarded $1,195,762 over two years to biotechnology company Valerion Therapeutics (formerly 4s3 Bioscience) for development of a treatment for myotubular myopathy (MTM)
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SBMA: Mixed Results in Study of Clenbuterol
May 29, 2013
A 20-person, open-label pilot trial conducted in Italy and designed to test the safety, tolerability and efficacy of clenbuterol in people with spinal-bulbar muscular atrophy (SBMA, or Kennedy disease) found that the drug was well-tolerated, although some possible safety is
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Moving Toward 'Next-Generation' Gene Therapy
May 22, 2013
Planning for the next generation of gene and stem cell therapies for muscular dystrophies — even as the first generation is still under development — was the theme of a joint symposium sponsored by MDA and the Association Française Contre les Myopathies (French Association Against Myopathies, or AFM) at the 16th annual me
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More Data Reported from Neuralstem's Phase 1 Stem Cell Trial in ALS
May 22, 2013
Recently announced data from a phase 1 clinical trial of NSI-566 neural stem cells in people with amyotrophic lateral sclerosis (ALS) show that a subset of trial participants had better outcomes than the others.
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New MDA/ALS Center Opens at LSU
May 20, 2013
The Muscular Dystrophy Association has designated the MDA clinic at Louisiana State University Health Sciences Center School of Medicine in New Orleans as an MDA/ALS center. This brings the total number of these specialized clinics to 44.
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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, and that it appears to increase the activity of the gene for the frataxin protein.
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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 deficiency) to a
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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 United States. Enrollment is expected to begin soon.
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26th Annual MDA Ride for Life Breaks $1 Million Mark to Help Fight Muscle Disease
May 10, 2013
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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.
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DMD UPDATE: Drisapersen Outperforms Placebo in Walking Test
May 08, 2013
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