Follow-up gene therapy study for MPS IIIA participants
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This follow-up study looks at how a past gene therapy treatment (UX111) is affecting you over time. It mainly involves continued visits and safety checks, and it helps researchers understand long-term benefits and risks.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have already taken part in an earlier study where you received UX111
- A parent or legal guardian must be willing and able to review and sign consent
- You (and your caregiver) must be able to follow the study visit schedule
- You cannot be joining another study that could interfere with measuring UX111’s safety or effects
- You must not have any other health situation that makes the required study procedures impossible
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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