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 ...

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 ...

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 ...

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 ...

On March 30, 2026, the FDA approved a high dose regimen for Spinraza (nusinersen) for the treatment of spinal muscular atrophy.

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 ...

Some people will do anything for the ones they love… even if it means starting life all over again at 72. When Zhu Yunchang’s ...

Credit: Novartis. The approval was supported by data from phase 3 STEER study, which enrolled patients with SMA who were treatment-naive and able to sit but never able to walk independently. Itvisma ...

Editor’s note: This is an automatically generated transcript. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription. So most recently, there has been the ...

Children born with a rare neuromuscular disorder are being given another chance in life thanks to a new treatment experts are ...

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.