Repurposing drug in Duchenne muscular dystrophy
Duchenne Muscular Dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact. DMD is one of four conditions known as dystrophinopathies. In Europe and North America, the prevalence of DMD is approximately 6 per 100,000 individuals.
Alport syndrome is a genetic condition characterized by kidney disease, hearing loss, and eye abnormalities. Alport syndrome is estimated to affect approximately 1 in 5,000-10,000 people in the general population.
Preclinical molecule development.