Infants with spinal muscular atrophy (SMA) who are presymptomatic and given the gene therapy Zolgensma in the first six weeks of life have better motor, respiratory, and nutritional outcomes, ...

Zolgensma gene therapy is the most effective in infants with SMA who are presymptomatic and 6 weeks old or younger, according ...

Variations in a DNA building block of genes tied to processes like inflammation associated in a study with disease type, ...

Scientists sought to understand the frequency of severe respiratory tract infections in SMA children, notably those needing ...

Columnist Halsey Blocher considers how her visible disability affects the way people treat her. That's the power of ...

Oral functions were found to be diminished in adults with spinal muscular atrophy in three out of five tests in a new study.

Treatment with Spinraza (nusinersen) can help make walking less tiring over time for ambulatory patients with SMA, a study found.

Heartbreak and resilience are common themes columnist Helen Baldwin has observed after Hurricane Helene and when her third ...