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 ...

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

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

Spinal muscular atrophy (SMA) and spina bifida are distinct conditions that affect the nerves that allow for voluntary motor control. Share on Pinterest press coverage photography/Getty Images SMA is ...

Biogen (BIIB) just cleared two important milestones, with FDA approval of a new High Dose Regimen for SPINRAZA in spinal muscular atrophy and positive Phase 2 data for litifilimab in cutaneous lupus ...

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 ...

"Managing pain in patients with SMA begins with the recognition that there are predictable etiologies leading to pain in SMA and investigating them during each medical visit is important to implement ...

US regulators have approved a high-dose version of Biogen Inc.’s drug for a rare muscle disorder, giving the company a boost as it competes with a gene therapy from Novartis AG.

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 ...

Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. So most recently, there has been the ...

Campaigners hope pilot will result in heel prick test for rare condition being approved across Britain ...