Sma - clinical

The use of SMN replacement therapies (nusinersen and onasemnogene) is dramatically altering the natural disease history of spinal muscular atrophy (SMA). Due to maximal benefit with early treatment, the American College of Obstetrics and Gynecology (ACOG) recommended that SMA be included on universal carrier screening for all pregnant women beginning 03/2017 and was added to RUSP recommendations for newborn screening (NBS) on 07/2018. With implementation of prenatal diagnosis and NBS for SMA, the goal is earlier treatment initiation and better clinical outcomes.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research