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 of life have better motor, respiratory, and nutritional outcomes, ...

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

The finding differs from the typical SMA type 0 progression, where symptoms manifest in the womb and untreated babies survive ...

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.

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

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

For columnist Jasmine Ramos, finding accessible volunteer opportunities has strengthened her sense of purpose and passion for ...

NIDO-361, a treatment for spinal and bulbar muscular atrophy (SBMA) now in a Phase 2 trial, was granted orphan drug ...

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, ...

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