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 ...

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 ...

Long-term data from DEVOTE/ONWARD studies show benefits of high dose nusinersen in people living with spinal muscular atrophy (SMA)New Phase 1b data further illustrate potential of salanersen in SMA, ...

"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 ...

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 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 ...

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 ...

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 ...

In Trumbull County, a local nonprofit will once again be hiding colorful candy-filled eggs in yards this Easter to help a ...

Building on the recent U.S. FDA Fast Track Designation for AJ201 (Rosolutamide) and encouraging Phase 2 clinical results, AnnJi presented two scientific abstracts spanning translational transcriptomic ...