A Litchfield woman is running the Boston Marathon to raise awareness for spinal muscular atrophy and honor her daughter, who defied medical expectations by living beyond her second birthday.

CUMBERLAND COUNTY, Pa. — Nicole Moore of Cumberland County is accomplishing goals she previously thought were impossible. Thanks to a revolutionary at-home treatment, she’s becoming more independent ...

Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

Children born with a rare neuromuscular disorder are being given another chance in life thanks to a new treatment experts are ...

Spinal muscular atrophy is a genetically inherited disorder that causes muscle weakness. Adults can get spinal muscular atrophy, but it’s rare in adults and progresses slowly. It doesn’t typically ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular ...