Plain-English translation of NCT04174157 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
This is a long-term tracking study for people with spinal muscular atrophy (SMA) who are being treated with OAV-101 (). Rather than testing a new drug, this study simply follows patients over 15 years to understand how they're doing, what treatments they receive, and how the medication affects their health over time. This information helps doctors and researchers learn more about living with SMA.
Spinal muscular atrophy is a rare condition, and doctors need to understand how this new gene therapy treatment works over the long term in real-world settings. This registry collects real-world information that can help improve care and understanding of the disease.
You likely qualify if…
You likely don't qualify if…
You will continue to receive your normal medical care from your regular doctor — nothing changes in how you're treated. Researchers will simply follow your progress and collect information about your health, treatments, and how you're doing over the next 15 years. There are no additional visits or tests required beyond what you're already doing with your care team.
AI-generated summary from trial data · Sep 24, 2026 · Not medical advice
United States