Research

posted on February 2, 2012 - 4:55pm
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-length DUX4 protein disrupts numerous biochemical pathways that normally would help muscle cells survive, mature and develop specialized roles.
posted on February 1, 2012 - 10:00am
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 for, the more than 40 neuromuscular diseases in its program. MDA's Board of Directors reviewed and approved the new grants based on recommendations from the Association's Scientific and Medical...
posted on January 27, 2012 - 6:00am
The U.S. Transportation Security Administration (TSA) recently launched a new program designed to help people with disabilities and medical conditions fly on commercial airlines with a minimum of hassle. TSA is the arm of the U.S. Department of Homeland Security, and is best known for its uniformed officers who staff security checkpoints at airports and inspect travelers, their baggage and...
posted on January 25, 2012 - 6:00am
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 have been receiving the drug through its expanded access program. Enrollment is by invitation only.
posted on January 19, 2012 - 5:09pm
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 finding that helped explain the molecular basis of facioscapulohumeral muscular dystrophy (FSHD).
posted on January 16, 2012 - 6:00am
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 rejection of enzyme replacement therapy in Pompe disease (acid maltase deficiency), a new study has shown.
posted on January 13, 2012 - 2:05pm
MDA has awarded $1 million to Tivorsan Pharmaceuticals for development of TVN-102, an experimental treatment for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).
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