Biogen’s successor to Spinraza (nusinersen) has shown benefit in spinal muscular atrophy (SMA) patients who had suboptimal clinical status despite prior administration of gene therapy. In the Phase Ib ...

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 ...

Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies ...

SMA is a genetic condition that impacts motor neurons, leading to muscle weakness that affects essential functions like breathing and movement. Treatments like Spinraza and Zolgensma can help manage ...

"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 ...

Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, ...

The jury’s still out. In a survey of state newborn screening programs for spinal muscular atrophy, researchers found that testing and tracking protocols varied among states, and that patients often ...

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 ...

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 ...