microdystrophin

posted on September 1, 2008 - 10:07am
Real progress is being made in supplying functional dystrophin genes to treat Duchenne muscular dystrophy (DMD), a disease in which mutated dystrophin genes keep this critical protein from being produced in muscle fibers.
posted on July 1, 2006 - 2:41pm
QUEST Vol. 13, No. 4
FDA approves Myozyme for Pompe disease