Overview
The primary objectives of the study are to prospectively evaluate pregnancy complications and outcomes in participants with SMA, birth outcomes and adverse effects in infants born to participants with SMA, who were exposed to nusinersen up to 14 months prior to the first day of their last menstrual period (LMP) before conception, 14.5 months before the date of conception, and/or at any time during their pregnancy. The secondary objective of the study is to evaluate pregnancy outcomes in participants with SMA exposed to nusinersen as compared with participants without SMA who were not exposed to nusinersen (e.g., participants from external, general population comparators).
Eligibility
Key Inclusion Criteria:
- Participant who is currently pregnant (or was pregnant during the relevant exposure window) and enrolled in the United Kingdom (UK), United States (US), or Italy network of the ISMAR registry or the Germany, Austria, or Switzerland network of the SMArtCARE registry
- Genetic documentation of spinal muscular atrophy linked to chromosome 5q (5q SMA)
- Documentation that the participant was exposed to nusinersen up to 14 months prior to the first day of their LMP before conception, 14.5 months before conception, and/or at any time during their pregnancy
Key Exclusion Criteria:
- Treatment with risdiplam at any time from the first day of their LMP, 2 weeks prior to the date of conception (approximately 5 half-lives), and/or plans to receive treatment with risdiplam during pregnancy
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply