St. Jude Children's Research Hospital
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.
Primary Objectives * Describe maternal-fetal monitoring and pregnancy outcomes from instances of healthy women dosed with risdiplam to treat their fetus diagnosed with Spinal Muscular Atrophy (SMA). * Describe delivery outcomes associated with prenatally treated SMA. Secondary Objectives * Describe postnatal clinical outcomes of women that were dosed with risdiplam during pregnancy to treat an affected fetus. * Describe clinical outcomes of children with SMA treated prenatally. This is a longitudinal retrospective/prospective registry study with the expected sample size of 30 mother-child…
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.
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