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(R) (nusinersen) for the treatment ...
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 ...
On March 30, 2026, the FDA approved a high dose regimen for Spinraza (nusinersen) for the treatment of spinal muscular atrophy.
Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of children and adults with ...
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 ...
Biogen Inc. BIIB shares are up on Monday following the FDA’s approval of a new high-dose regimen for Spinraza (nusinersen), a ...
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 ...
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 main beneficiary of Roche’s discontinuation of an investigational spinal muscular atrophy drug is Scholar Rock, which was ...
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 ...
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 ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results