Spinal muscular atrophy explained after Jesy Nelson reveals her twins’ diagnosis - The singer shared an emotional update ...

Risdiplam, an oral pre–messenger RNA splicing modifier, is an efficacious treatment for persons with symptomatic spinal muscular atrophy (SMA). The safety and efficacy of risdiplam in presymptomatic ...

University of Pittsburgh School of Medicine researchers carrying out a small pilot clinical trial demonstrating that a drug-free, minimally invasive intervention targets the root cause of progressive ...

Distal spinal muscular atrophy (DSMA) is a rare genetic disease that causes a loss of muscle movement. It affects muscles further away from the center of the body, such as the hands, feet, and legs.