Infants with SMA type 1 born since 2016 experience better functional outcomes and life expectancy with disease-modifying treatments.

New FDA-approved human clinical trials are beginning for a drug that could give a teenager the opportunity to speak for the first time. A Sacramento-area family is calling it a miracle.

Liver issues, including a buildup of fat in the liver, occur before the onset of neuromuscular disease, per a study in a mouse model of SMA.