Treatment with salanersen slowed neurodegeneration and improved motor function in children with SMA in a Phase 1 trial, interim results show.

In perhaps a first in the country, a newborn found carrying the SMN1 gene mutation for Spinal Muscular Atrophy (SMA) through prenatal genetic screening is being administered presymptomatic ...

In the SUNFISH study, statistically significant improvement (1.55 point mean difference; P = .0156) in motor function was observed in treated patients at 12 months (1.36 points; 95% CI, 0.61-2.11 ...

In our pursuit to advance healthcare, some rare diseases still go unnoticed, with most people unaware of their seriousness. One such condition is Spinal Muscular Atrophy (SMA), a genetic disorder ...

Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.

Spinal Muscular Atrophy (SMA) is a common genetic disorder affecting both children and adults, frequently encountered by pediatricians, neurologists, physiotherapists, and the general public.

Spinal muscular atrophy (SMA) is a severe neurological disease caused by reduced survival of motor neuron (SMN) protein levels. There is presently no cure for the condition, although current ...

An international study has discovered that people living with the neurodegenerative condition Spinal Muscular Atrophy (SMA) are at higher risk of developing fatty liver disease, suggesting that ...

Spinal muscular atrophy (SMA), a rare genetic disease that affects the nervous system and muscles, is caused by mutations in the SMN1 gene. The SMN1 gene encodes for the SMN protein, which is ...

Treatment with nusinersen was linked to increased motor function and reduction of the neurodegenerative biomarker neurofilament light chain in children with spinal muscular atrophy, according to ...

Neurology Source Reference: Crawford TO, et al "Safety and efficacy of apitegromab in patients with spinal muscular atrophy types 2 and 3" Neurology 2024; DOI: 10.1212/WNL.000000000020915.

The most efficient one called SMN 1, that’s what’s missing in spinal muscular atrophy,” Tilton said. Now, the first FDA-approved oral medication to treat SMA, Evrysdi, can turn on the second ...