A case report described an infant with concurrent HDFN and SMA who was succesfully treated with an onasemnogene abeparvovec ...
Sales growth driven by continued strong performance from Entresto (+31% cc), Cosentyx (+25% cc), Kesimpta (+49% cc), Kisqali (+49% cc), Pluvicto (+42% cc) and Leqvio (+114% cc) Core operating income ...
Background: Spinal and bulbar muscular atrophy (SBMA) or Kennedy disease [OMIM ... consent form and allowed anonymised publication of their clinical information and photos. The study included all ...
A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully ...
Three patients with spinal muscular atrophy had improved muscle strength and could walk farther after a month of daily spinal ...
A new drug-free, minimally invasive intervention targets the root cause of progressive loss of neural function in spinal muscle atrophy (SMA), an inherited neuromuscular disease.