A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its partner protein, dystrobrevin, offering new pathways for ...
PTPN1/2 inhibition promotes muscle stem cell differentiation in Duchenne muscular dystrophy. Life Science Alliance , 2024; 8 (1): e202402831 DOI: 10.26508/lsa.202402831 Cite This Page : ...
Solid Biosciences advances gene therapies for muscular and cardiac diseases, gaining FDA clearance for SGT-212 and targeting ...
Duchenne Muscular Dystrophy (DMD) is a rare progressive disorder. People with DMD are missing muscle-protecting protein. This is due to a fault in the gene producing dystrophin. Muscles to become ...
Prenatal diagnostic testing can now determine whether a fetus carries a debilitating or fatal sex-linked mutation. But with such screening, why hasn't the disease allele frequency gone down ...
Deramiocel is a cell therapy that has healing effects in muscle cells. If approved, deramiocel would be a once-quarterly ...
This year’s top stories on Duchenne muscular dystrophy (DMD) managed care included a novel imaging method for tracking disease progression, an FDA approval of the first nonsteroidal therapy ...
Sarepta Therapeutics has decided not to continue the development of vesleteplirsen, a follow-up to its Duchenne muscular dystrophy (DMD) therapy Exondys 51. The drug – formerly known as SRP-5051 ...
A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. McGill University researchers have discovered that an ...
Solid Biosciences ended Q3 2024 with $171.1 million in cash, securing operations through 2026. Initial Phase 1/2 trial results for SGT-003 in DMD expected in Q1 2025. Wedbush initiated coverage on ...
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