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 ...

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 ...

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

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

A UK study found more than 80% of 80 SMA type 1 children on a DMT had scoliosis or other abnormal spinal curvature needing ...

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

The U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of Evrysdi (risdiplam), an approved oral ...

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

New 5mg tablet offers the same established efficacy and safety as the Evrysdi oral solution for individuals living with spinal muscular atrophy.

We think Biogen's neurology, immunology, and focus on rare diseases support a narrow moat. Biogen's strategy has its roots in the 2003 merger of Biogen (Avonex for treating multiple sclerosis, or MS) ...