NCT07805278
A Registry of Cases of Spinal Muscular Atrophy
Recruiting · Not specified · St. Jude Children's Research Hospital · registry updated 2026-09-30
Inclusion and exclusion lines below are quoted from ClinicalTrials.gov. No match score is shown, because a score needs a person's age, biomarkers, and treatment dates. Confirm the record with the study team.
Because no long-term record of prenatal Spinal Muscular Atrophy (SMA) treatment exists, this study will address an important gap in the understanding of the safety and benefit of treatment while in the womb. The registry will help guide best practices for monitoring pregnant women and their fetus with SMA, if treated with a medication while in the womb, and to follow the baby after birth for signs of safety and added benefit birth and support future research and study development.
Inclusion
- Current or past pregnancy during which the fetus received a genetic diagnosis of SMA.
- Current or past treatment with SMA-directed therapy intended to treat the fetus during gestation (such as risdiplam, nusinersen, onasemnogene abeparvovec, or another product intended to treat SMA).
Exclusion
- Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.