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A Registry of Cases of Spinal Muscular Atrophy

A Registry of Cases of Spinal Muscular Atrophy Diagnosed in the Prenatal Period That Received Disease-Modifying Therapy

Who can join

All ages · All sexes

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.

About the study

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.

Sponsor: St. Jude Children's Research Hospital · Participants: 30 · Started: 2026-09

Contact the study team

• Richard Finkel, MD · Phone: 888-226-4343

Official record on ClinicalTrials.gov — NCT07805278

Locations in the U.S.

Conditions

• Spinal Muscular Atrophy

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.