Registry of Spinal Muscular Atrophy Diagnosed Before Birth and Treated With Medication
Follows pregnancies and babies with spinal muscular atrophy treated before birth.
Plain-language title generated from the registry record
Registry title: A Registry of Cases of Spinal Muscular Atrophy
Key facts
- Where
- Memphis, United StatesSee the site
Where in United States
- St. Jude Children's Research HospitalMemphis, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- St. Jude Children's Research Hospital
- Trial code
- NCT07805278
- Record updated
- 22 October 2026
- Original description
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.Read full original descriptionShow less
A Registry of Cases of Spinal Muscular Atrophy Diagnosed in the Prenatal Period That Received Disease-Modifying Therapy
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.