The U.S. Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for ...

On this episode, an infant with a harrowing diagnosis defies all odds after receiving a $2 million genetic treatment. Then, a ...

A girl on Long Island is speaking out after a clinical trial for Spinal Muscular Atrophy was cut by President Trump ...

Hi friends! Shane here! I just wanted to quick put up some reflections on my day today. I got my 7th Spinraza injection and ...

The FDA on Wednesday approved the industry’s first-ever tablet treatment for spinal muscular atrophy— Roche ’s SMN2 splicing ...

How a tiny spinal implant could help those with muscle-wasting disease - Three people with a muscle-destroying disease got ...

Three patients with spinal muscular atrophy had improved muscle strength and could walk farther after a month of daily spinal ...

Evrysdi is a non-invasive, disease-modifying option that can be swallowed whole or dispersed in water for patients with ...

The Muscular Dystrophy Association (MDA) today launched the nation’s largest St. Patrick’s Day fundraising campaign, MDA Shamrocks, as part of its celebration of 75 years of progress and impact in ...

Genentech and Roche are investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s ...

A new spinal cord implant may help people with spinal muscular atrophy (SMA) regain some muscle function, giving them ...

The FDA has approved Evrysdi in tablet form for patients with spinal muscular atrophy aged 2 years and older who weigh more ...