Clinical Trial: Service Evaluation for Newborn Screening for Spinal Muscular Atrophy

U.S., Oct. 23 -- ClinicalTrials.gov registry received information related to the study (NCT07888868) titled 'Service Evaluation for Newborn Screening for Spinal Muscular Atrophy' on Aug. 07.

Brief Summary: Spinal Muscular Atrophy (SMA) is a disease that causes progressive muscle weakness. There are many types- the most severe and most common, type 1, affects very young babies, and is life-threatening if untreated. Other types can cause significant disability. Most cases are caused by a missing part of the survival motor neuron 1 (SMN1) gene. Another related gene, SMN2, can partially compensate for this affected gene. With more copies of SMN2 broadly leading to a milder disease.

Three new treatments can slow or prevent symptoms, but they w...