Regina Trollmann, MD, of the Division of Pediatric Neurology, Department of Pediatrics, Friedrich-Alexander-University of Erlangen-Nürnberg, Erlangen, Germany, and colleagues, did a retrospective ...

Emma Ciafaloni, MD, FAAN, explores the groundbreaking approval of onasemnogene abeparvovec-xioi for spinal muscular atrophy and its impact on the treatment paradigm for this rare disease. This is a ...

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

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 ...

The FDA has approved a higher-dose regimen of Spinraza for spinal muscular atrophy, providing a new treatment option for patients. The regimen includes 50 milligram per 5 milliliter and 28 milligram ...

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 ...

Cure SMA, the leading nonprofit organization supporting those impacted by spinal muscular atrophy (SMA), celebrates that Biogen today announced FDA approval of the High Dose regimen of SPINRAZA(TM) ...

Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

The approval was anchored on data from the pivotal DEVOTE study that investigated the efficacy and safety of the High Dose Regimen of SPINRAZA in ...

The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to the mother during pregnancy and to the child after birth. More than 2 years ...

Scotland is now screening every newborn for a condition that can kill within two years. Here is what parents across the UK need to know.