Spinal Muscular Atrophy

Ongoing follow-up in the START phase 1 study supported “the long-term favorable safety profile” of Zolgensma in children aged up to 6 years with spinal muscular atrophy type 1, according to findings published in JAMA Neurology.

Evrysdi, a survival motor neuron 2-directed RNA splicing modifier, both sustained and improved motor function at 24 months in children and adults with type 2 or type 3 spinal muscular atrophy, according to results from the SUNFISH trial.

Progress in the field of gene and cell therapy continues with rapid development despite the negative impact of COVID-19 on research into novel treatments, according to the director of FDA’s Center for Biologics Evaluation and Research.

The FDA approved risdiplam for patients aged at least 2 months with spinal muscular atrophy, according to an agency press release.