Spinal muscular atrophy is caused by mutations in the survival motor neurons gene, SMN1. The SMN protein localizes to both the cytoplasm and the nuclear bodies. One hallmark of spinal muscular ...

A child with a rare genetic disorder — spinal muscular atrophy type I — was treated prenatally for the first time. Courtesy of Jakob Owens  via Unsplash During an amniocentesis test, a long, thin ...

When the SMN1 gene is mutated, the body cannot make enough SMN protein, and the nerves cannot adequately send out signals to muscles. As a result, the muscles — especially in the thighs ...

Had the agency known about the manipulation, it would have delayed the approval of Zolgensma, Wilson Bryan, the director of the FDA division that reviewed Novartis’s gene therapy, tells ST AT. The ...

Tilton. Now, the first FDA-approved oral medication to treat SMA—Evrysdi—can turn on the second SMN gene, making it more ...