The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric ...

The US Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for people living with spinal muscular atrophy (SMA).

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...

Ionis relies on upfront payments and licensing fees from partners, as well as Spinraza royalties, to drive revenue today, but its rapidly advancing portfolio should help diversify revenue. 2025 is ...

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

Exercise can make a significant difference for patients with SMA by working alongside other medical advancements to improve ...

Discover the first in-utero treatment for spinal muscular atrophy (SMA) using risdiplam. Promising results show no SMA signs ...

Spinal muscle atrophyorspinal muscular atrophy(SMA) is a genetic disorder that can affect babies, children, and adults. A ...

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

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 ...

Researchers have developed a regenerative medicine method that successfully grafts myoblasts onto healthy muscle, boosting ...