Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...

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

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

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

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

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

Scientists report results from a promising new approach to treat the rare neurodegenerative disorder. Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before ...

Evrysdi is a non-invasive, disease-modifying option that can be swallowed whole or dispersed in water for patients with spinal muscular atrophy. The FDA has approved Roche’s Evrysdi (risdiplam) tablet ...

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

Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to ...