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 ...

Patients with Spinal Muscular Atrophy urge the Prime Minister for access to affordable generic Risdiplam to save lives.

"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 ...

A multidisciplinary team at Medcare delivered the therapy, led by its Gene Therapy Unit clinical lead, Dr. Vivek Mundada, ...

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 ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

"It's encouraging and it gives me hope that they're finally understanding that we deserve to have treatment just like the rest and that we deserve a chance," she said. "At least a ...

Spinal muscular atrophy (SMA) is a severe neurological disease caused by reduced survival of motor neuron (SMN) protein levels. There is presently no cure for the condition, although current therapies ...

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 ...

The FDA approved a risdiplam tablet for SMA patients aged 2+ and over 44 lbs, matching the oral solution's efficacy and safety. Risdiplam, an SMN2 pre-mRNA splicing modifier, enhances SMN protein ...

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 ...

Ellie Kerrigan, who has spinal muscular atrophy, shared her journey through Alabama sorority recruitment on TikTok ...