Category Archives: News from Other Sources

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

Researchers are seeking participants for a study to identify accurate biological indicators of ALS onset, progression and response to treatment.

The Muscular Dystrophy Association (MDA) announced today it has awarded $1 million to Tivorsan Pharmaceuticals to help speed preclinical work vital to a filing of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration …

With funding from MDA, the ALS Therapy Development Institute is testing whether the immune-system modulator CTLA4-FC slows disease progression in ALS mice.

Precision delivery of human frataxin protein into cells favorably affects growth, life span and the heart in a mouse model of Friedreich’s ataxia.

The MDA-supported ALS Therapy Development Institute will collaborate with Biogen Idec and UCB to test CDP7657, a compound that modulates the immune system.

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

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

Enrollment is open for a trial of AT2220, a drug designed to enhance the effects of enzyme replacement therapy in people with acid maltase deficiency.

Enrollment is open for a trial of AT2220, a drug designed to enhance the effects of enzyme replacement therapy in people with acid maltase deficiency.