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 ...

Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers ...

This high dose nusinersen regimen for spinal muscular atrophy includes 50mg loading doses and 28mg maintenance doses.

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 ...

Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of children and adults with ...

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

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 ...

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 ...

Ashley Webb, MD, Program Director, Pediatric Neurology, at the University of Texas Health Science Center at Houston McGovern Medical School, discusses the need to make genetic therapy more accessible ...

The main beneficiary of Roche’s discontinuation of an investigational spinal muscular atrophy drug is Scholar Rock, which was ...

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.