In 2010, PTC Therapeutics released preliminary results of its phase 2b clinical trial for Duchenne muscular dystrophy, with participants not showing a significant improvement in the six minute walk distance after the 48 weeks of the trial.
Duchenne muscular dystrophy, a severe recessive X-chromosome linked form of muscular dystrophy
It is the story of Dusty, a 20-year-old young man with Duchenne muscular dystrophy.
alpha-dystroglycan is extracellular and binds to merosin alpha-2 laminin in the basement membrane, while beta-dystroglycan is a transmembrane protein and binds to dystrophin, which is a large rod-like cytoskeletal protein, absent in Duchenne muscular dystrophy patients.
Evidence is accumulating that suggests that osteopontin plays a number of roles in diseases of skeletal muscle, such as Duchenne muscular dystrophy.
The syntrophins are a family of five 60-kiloDalton proteins that are associated with dystrophin, the protein associated with Duchenne muscular dystrophy and Becker muscular dystrophy.
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Particularly successful recently has been the delivery of mesoangioblast cells into the Golden Retriever dog model of Duchenne muscular dystrophy, which effectively cured the disease.
Biostrophin, another drug against Duchenne muscular dystrophy