But for Brooklyn, who lives with spinal muscular atrophy, or SMA, a rare genetic condition that affects muscle strength and ...

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 ...

A Prescription Drug User Fee Act target date of September 22, 2025 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License ...

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 ...

SMA is a rare genetic disease that destroys motor neurons — the cells that control movement, swallowing, and breathing.

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 ...

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 ...

Please provide your email address to receive an email when new articles are posted on . An investigational, fully human monoclonal antibody developed as a muscle-targeted therapy for spinal muscular ...

Please provide your email address to receive an email when new articles are posted on . At the time of last follow up, 82.6% of children born with SMA type 1 from 2016 on were still alive. Of the 140 ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...