Muscle fibers in children with SMA type 1 show substantial differences in components of the myosin protein called heavy ...

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

Infants with spinal muscular atrophy (SMA) who are presymptomatic and given the gene therapy Zolgensma in the first six weeks ...

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

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

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 ...

Twin girls with spinal muscular atrophy (SMA) type 0 are still alive with normal motor function more than two years after treatment with Zolgensma (onasemnogene abeparvovec-xioi). This finding ...

Treatment with Spinraza (nusinersen) can help make walking less tiring over time for ambulatory patients with spinal muscular atrophy (SMA), a study reported. The study, “Performance fatigability in ...