The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in patients 2 years and older with confirmed mutation in ...

Modulating the activity of a kinase in motor neurons may help mitigate mitochondrial defects and other symptoms of spinal muscular atrophy, offering a new therapeutic avenue for the devastating ...

Former Little Mix star Jesy Nelson revealed her twin daughters, Ocean Jade and Story Monroe Nelson-Foster, had been diagnosed with Spinal Muscular Atrophy (SMA) Type 1, a rare genetic condition that ...