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Recruiting NCT07805278

A Registry of Cases of Spinal Muscular Atrophy

Conditions: Spinal Muscular Atrophy

Sex: All
Enrollment: 30
Sponsor: St. Jude Children's Research Hospital

Location: St. Jude Children's Research Hospital Memphis Tennessee

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.

Eligibility Criteria

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.

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT07805278). StuddyBuddy aggregates publicly available trial information.