A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies

To be included in this study, you need to be a pregnant woman (or have been pregnant recently) with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) that is linked to a specific change on chromosome 5q. You must also have received nusinersen (Spinraza) at some point before or during your pregnancy. This includes if you had the treatment up to 14 months before your last period before becoming pregnant, up to 14.5 months before you conceived, or at any time during your pregnancy. You should also be part of specific SMA research groups in the US, UK, Germany, Austria, or Switzerland.

You would not be able to join this study if you have been treated with another SMA medication called risdiplam during a specific timeframe around your pregnancy, or if you plan to use risdiplam during your pregnancy. The study is specifically looking at the effects of nusinersen, so having taken other SMA medications would make it difficult to understand just the effects of nusinersen.

If you decide to take part, the study will begin collecting information from you. You'll be contacted around every three months during your pregnancy to ask about your health and how your pregnancy is progressing. Your doctors will also be contacted during each trimester of your pregnancy, when you're about 6 or 7 months pregnant, and again about four weeks after your baby is born.

After your baby is born, your baby's doctors will be contacted when your baby is 1, 2, 6, 12, 18, and 24 months old to gather information about their health and development. You will be part of the study from the time you join until the end of your pregnancy and up to 12 weeks after your baby is delivered. Your baby will continue in the study for up to two years after their birth. Overall, the study is expected to run for at least 10 years.