The first prenatal therapy for spinal muscular atrophy (SMA) showed promising results, a case report indicated. More than 2 ...

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

Scientists at St. Jude Children’s Research Hospital report results from a promising new approach to treat the rare ...

February 19, 2025) Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists ...

PASIR MAS, Feb 19 (Bernama) -- "A mother's love knows no bounds, leads to heaven," are words that perfectly describe the ...

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 ...

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

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

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 ...

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