Disease Focus: Muscular Dystrophy
Stem Cell Therapy for Duchenne Muscular Dystrophy
At its July meeting, the CIRM governing Board was honored to hear from Caleb Sizemore, a delightful and courageous young man who was born with Duchenne muscular dystrophy, a fatal genetic disease that causes progressive degeneration and weakening of muscles. Many children with DMD don’t live past their 20s often due to faltering lung and […]
Spotlight on Muscular Dystrophy and Stem Cell Research – Helen Blau, Stanford
Duchenne muscular dystrophy (DMD) is the most severe form of muscular dystrophy that affects 1 in 3500 boys and leads to progressive muscle degeneration and death by the second decade of life. It’s been nearly thirty years ago that dystrophin, the mutated gene that causes DMD, was identified. Without the large structural protein encoded by […]