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

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

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.

Halsey Blocher is a young woman who was diagnosed with SMA Type 1 in 1999 after a blood test revealed a deletion of the survival motor neuron gene. She now lives with her loving family in Fort Wayne, ...

Ari Anderson resides in Charlotte, North Carolina, and was diagnosed with SMA type I at 6 months old in 1982. At the time, the prognosis was almost hopeless. Today’s medical therapies have changed the ...