Spinal muscular atrophy patient registry for OAV-101
Part of Brain & nervous system clinical trials.
This is a registry that collects information from people with spinal muscular atrophy (SMA) who have been treated with OAV-101. It helps researchers understand how patients do over time after this treatment.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a genetically confirmed diagnosis of spinal muscular atrophy (SMA).
- You have been treated with OAV-101 (Zolgensma).
- You (or your legal guardian) can provide consent, and if needed, you can agree as well (assent).
- You are not currently taking part in another SMA treatment study that uses an investigational drug.
- If you are on an expanded access/compassionate use program for OAV-101 (like MAP/EAP/SPI/NPP), you can still join.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study creates a registry and clinical hub for adults with 5q spinal muscular atrophy (SMA). It aims to track health outcomes and improve care for people living with SMA.
This trial tests a new medicine called EXG001-307 for babies with spinal muscular atrophy (SMA) types 1 and 2. The goal is to see if it is safe and helps with muscle strength and breathing.
This trial is a registry that collects health information from people with spinal muscular atrophy (SMA). It helps researchers understand the condition better and may support future studies and treatments.
This study follows the long-term safety of Zolgensma gene therapy in Brazilian children with spinal muscular atrophy (SMA). It helps doctors understand how safe the treatment is over time.
This study reviews medical records of people with spinal muscular atrophy type 1 who received the gene therapy Zolgensma (onasemnogene abeparvovec) to understand their treatment journey and outcomes. It may help doctors improve care for future patients.
This study looks at how safe and effective Zolgensma is when given as an injection into the spinal fluid. It's for people with spinal muscular atrophy who have already been treated with Zolgensma.
Hear when a new Spinal Muscular Atrophy (SMA) trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.