Muscular Dystrophies

An 11½-minute interview with researcher Stephen Tapscott explores recent findings about the molecular basis of facioscapulohumeral MD

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.

MDA has awarded 38 grants totaling more than $12 million to support research into general muscle health and more than 15 neuromuscular diseases in its program

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...

New research explains probable ways in which DUX4 protein, if made at the wrong time and in the wrong place, leads to FSH dystrophy

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).

MDA's $1 million grant will help fund the development of TVN-102, which attracts the muscle protein utrophin to the muscle-fiber membrane

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).

Molecular geneticist Stephen Wilton discusses his group's development of exon-skipping compounds to restore dystrophin production in Duchenne MD

posted on January 11, 2012 - 2:01pm
A January 2012 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores the potential of a strategy called exon skipping as a treatment for Duchenne muscular dystrophy (DMD). The podcast, which runs approximately 12 minutes, is part of a Nationwide Children's series called "This Month in Muscular Dystrophy."

MDA has awarded $750,000 to Summit Corp. to test a new formulation of its utrophin booster SMT C1100 for Duchenne muscular dystrophy

posted on December 20, 2011 - 12:05pm
MDA has awarded $750,000 to Summit Corporation PLC for development and testing of SMT C1100, the company's experimental drug for treatment of Duchenne muscular dystrophy (DMD). Summit is an Oxford, United Kingdom, drug discovery company. The award was made through MDA's Venture Philanthropy (MVP) arm, a part of MDA's translational research program.

Biologist Denis Guttridge discusses his team's work developing an NF-kappa B blocker as a potential treatment for Duchenne MD

posted on December 14, 2011 - 3:56pm
A December 2011 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores how an inhibitor of a protein called NF-kappa B has been beneficial in a mouse model of Duchenne muscular dystrophy (DMD) and is now being developed as a potential DMD treatment. The podcast is part of a Nationwide Children's series called "This Month in Muscular Dystrophy."

IDMC-8, held Nov. 30-Dec. 3, 2011, explored disease mechanisms, clinical aspects and ideas for future therapies in myotonic muscular dystrophy

posted on December 8, 2011 - 3:05pm
The 8th International Myotonic Dystrophy Consortium Meeting (IDMC-8), was an exciting mix of the latest scientific developments and clinical research in types 1 and 2 myotonic dystrophy (MMD1 and MMD1, also known as DM1 and DM2).
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