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

Advanced genetic testing helped clinicians identify novel SMN1 mutations in two children in China, and correctly diagnose ...

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

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 ...

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

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 ...