A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration.
McGill UniversityDec 16 2024 A novel drug holds promise for treating Duchenne muscular dystrophy , a rare genetic disorder that causes severe muscle degeneration.
By strengthening muscle repair rather than just slowing degeneration, therapies that stimulate muscle stem cell function have the potential to improve quality of life for DMD patients. It may help restore muscle function and, ultimately, offer greater independence." Building stronger muscles from stem cells Biotechnology company Kanyr Pharma originally developed the drug for cancer and metabolic diseases, but it has not yet been approved for any specific use.
Related StoriesUnlike gene therapy, which targets specific genetic mutations and isn't suitable for all patients, K884 works at the cellular level, restoring muscle repair regardless of the mutation causing the disease. This makes it a potential treatment option for all DMD patients, she added.
Muscular Dystrophy Cell Compound Genetic Genetic Disorder Life Science Muscle Research Stem Cells Wheelchair
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