Research News
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March 23, 2012
Investigators at the University of Michigan in Ann Arbor are conducting a study of the progression of centronuclear myopathies (CNMs), including the type known as myotubular myopathy (MTM); central
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March 19, 2012
More than 500 physicians, allied health care professionals and MDA staff attended the MDA's 2012 Clinical Conference in Las Vegas, March 4-7.
The program emphasized:
core information about genetics
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March 15, 2012
The biopharmaceutical company Repligen Corp., headquartered in Waltham, Mass., has launched a phase 1 clinical trial to test its experimental compound, RG2833, in adults with Friedreich's ataxia (FA
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March 14, 2012
Below are highlights of two recent studies in spinal muscular atrophy (SMA), a disease in which the nerve cells that control muscles (motor neurons) in the spinal cord die, causing progressive
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March 14, 2012
A March 2012 podcast from Nationwide Children's Hospital in Columbus, Ohio, presents the results of a phase 1 trial of gene therapy for gamma-sarcoglycan-deficientlimb-girdle muscular dystrophy (LGMD
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March 11, 2012
In type 1 myotonic dystrophy (MMD1, or DM1), expansions of DNA on chromosome 19 known as CTG repeats are converted to expansions in RNA called CUG repeats, which are toxic to nerve and muscles cells
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March 06, 2012
The Muscular Dystrophy Association has given a $300,000 grant to the Center for Genetic Medicine Research (CGMR) at Children’s National Medical Center in Washington, D.C., to continue standardized
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March 04, 2012
MDA has extended its strategic research partnership with the ALS Therapy Development Institute (ALS TDI) through 2012 and awarded a $2 million grant to support the nonprofit biotech's continued
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March 02, 2012
Update 5/21/12: A podcast on this topic is now available; see Podcast Explores Newborn Screening for DMD.
Researchers at Nationwide Children’s Hospital in Columbus, Ohio, working with the DNA
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February 29, 2012
Current trends and practices in the treatment of neuromuscular disease, and exciting new therapeutic developments will be the focus of MDA’s 2012 Clinical Conference March 4-7, at the South Point
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February 29, 2012
Researchers at Baylor College of Medicine in Houston and Isis Pharmaceuticals in Carlsbad, Calif., have announced encouraging results for their antisense-based strategy in development for the
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February 24, 2012
Below are highlights of two recent studies in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).
Loss of MSP protein a possible mechanism underlying ALS
Major sperm protein (MSP) appears
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February 23, 2012
Editor's note 3/15/12: This story was updated to reflect the availability of a podcast with Matthew Disney.
Small, laboratory-designed molecules can make a big difference in cells carrying the
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February 14, 2012
A phase 2 clinical trial is set to begin of TDI 132, a compound that modulates the immune system, the ALS Therapy Development Institute (ALS TDI) announced today.
TDI 132, also known as fingolimod (
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February 10, 2012
Duplications (extra copies) of the SMN1 gene are a "major" risk factor for developing sporadic (noninherited) ALS (amyotrophic lateral sclerosis), a team of scientists based in the Netherlands and
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February 08, 2012
Shortened versions of the muscle protein dystrophin— produced by skipping a section of genetic instructions called exon 51— appear to be functional, says a new report from the United Kingdom.
The
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February 02, 2012
A protein called DUX4, inappropriately produced ("expressed") in skeletal muscle fibers, is emerging as a major factor in facioscapulohumeral muscular dystrophy (FSHD).
In FSHD-affected muscles, full
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February 01, 2012
The Muscular Dystrophy Association has awarded 38 new grants totaling more than $12 million to fund research projects focused on its continuing mission to uncover the causes of, and develop therapies
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February 01, 2012
Six new MDA grants totaling $2 million have been awarded to research projects seeking to uncover the basic mechanisms that drive ALS (amyotrophic lateral sclerosis), with an eye toward the
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January 24, 2012
Jacobus Pharmaceutical of Princeton, N.J., has opened a randomized, placebo-controlled study of the drug 3,4-diaminopyridine (3,4-DAP) in 30 adults with Lambert-Eaton myasthenic syndrome (LEMS) who
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January 23, 2012
Preliminary data reported Jan. 17, 2012, by BrainStorm Cell Therapeutics demonstrate that the biotechnology company's experimental NurOwn stem cell technology has not caused any significant adverse
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January 19, 2012
Editor's note 2/2/12:This story was updated to reflect the availability of a podcast in which Stephen Tapscott is interviewed.
A little over a year ago, a team of researchers announced a crucial new
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January 18, 2012
The Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) is actively seeking participants for a study designed to identify and validate biomarkers in amyotrophic lateral sclerosis (ALS).
A
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January 15, 2012
Editor's note 2/2/12:This story was updated to reflect the award of a new MDA grant to Eric Sjoberg at Amicus Therapeutics.
Drugs that suppress the immune system can successfully prevent or reverse
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January 13, 2012
Editor's note 2/15/12: On Feb. 15, 2015, Justin Fallon and colleagues announced in the Journal of Neuroscience that they have identified an additional role for biglycan; namely, that it stabilizes
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