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 ...

Risdiplam works by targeting the SMN2 gene to induce increased production of the survival motor neuron (SMN) protein; a deficiency of this protein is what causes the most common forms of SMA.