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 ...

Discover how a 10-year-old boy with spinal muscular atrophy finds freedom and strength through swimming, inspiring others to defy physical limits. Read more at straitstimes.com. Read more at ...

I feel like my whole life has done a 360,” says Jesy Nelson in the final five minutes of Jesy Nelson: Life After Little Mix (Prime Video), the six-part documentary charting her life since leaving ...

Jesy Nelson broke down in tears after her campaign for more health checks on babies reached 100,000 signatures - meaning it ...

A positive newborn screening for spinal muscular atrophy (SMA) is currently considered a medical emergency. Without early treatment, severe disability or death in infancy are likely. However, research ...

"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 ...

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 ...

People with spinal muscle atrophy (SMA), an inherited neuromuscular disease, usually experience muscle weakness that impacts movement. New research suggests that electrical spinal cord stimulation ...

Infantile spinal muscular atrophy (SMA) is a neuromuscular disorder that affects the nerve cells in the spinal cord. It causes muscles to weaken and limits muscle movement. SMA is a rare condition.

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 ...

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 ...

"The benefits of newborn screening identified in the study presented here encompass not only the achievement of motor milestones, but also a reduced need for ventilator support or tube feeding and a ...