Scholar Rock Holding has resubmitted its application for its spinal muscular atrophy treatment to the Food and Drug Administration. The biopharmaceutical company said that it has sent in a new ...

The Muscular Dystrophy Association (MDA) celebrates today’s announcement that the U.S. Food and Drug Administration (FDA) has approved a High Dose Regimen of SPINRAZA® (nusinersen) for the treatment ...

Columbia researchers have discovered how a genetic defect leads to spinal muscular atrophy (SMA), a critical piece of information about the disease that neurologists have been seeking for decades. The ...

Spinal muscular atrophy (SMA) is a severe neurological disease caused by reduced survival of motor neuron (SMN) protein levels. There is presently no cure for the condition, although current therapies ...

Oral risdiplam (Evrysdi, Genentech) started in the first 6 weeks of life let most infants with presymptomatic spinal muscular atrophy (SMA) reach motor milestones typical of healthy babies, results of ...

Spinal and bulbar muscular atrophy (SBMA) is a rare inherited disease that causes progressive muscle weakness and wasting in ...

Biogen Inc. BIIB shares are up on Monday following the FDA’s approval of a new high-dose regimen for Spinraza (nusinersen), a ...

Distal spinal muscular atrophy (DSMA) is a rare genetic disease that causes a loss of muscle movement. It affects muscles further away from the center of the body, such as the hands, feet, and legs.

Patients with SMA often suffer from impaired bulbar function, which negatively affects quality of life and can be difficult to treat effectively. A German study prospectively followed children with ...

MIAMI, July 27, 2023 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of ...