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RECRUITING
Updated: Sep 8, 2026
A Registry of Cases of Spinal Muscular Atrophy
Brief Summary
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.
Key Information
Participation Criteria
Locations & Contacts
Inclusion Criteria:
- 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 Criteria:
- Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.