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 ...

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

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

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

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

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

The Muscular Dystrophy Association celebrates this new more accessible treatment option for people living with SMA. MDA - New ...

The 5 mg risdiplam tablet provides the same efficacy and safety for spinal muscular atrophy as the currently available oral ...

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug ...

Emma Larson's life was saved by a miracle drug. Those stories could cease to exist if NIH funding cuts proposed by DOGE go ...