Long-term monitoring for MPS IIIA treatment
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This study follows people with MPS IIIA (Sanfilippo syndrome type A) who have received or plan to receive the gene therapy rebisufligene etisparvovec, to monitor their health and treatment effects over time.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed diagnosis of MPS IIIA (Sanfilippo syndrome type A).
- You have received rebisufligene etisparvovec in a previous clinical trial or in regular medical care, or you plan to receive it as part of regular care.
- You agree to participate and sign a consent form (or a legal guardian signs for you if you are a minor or have cognitive limitations).
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 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.
This trial tests a gene-transfer treatment (delivered into the spinal fluid) for children with MPS IIIA, a rare genetic condition. It aims to improve or slow down brain and developmental changes caused by a missing enzyme.
This study tests whether a cannabis-derived medicine called cannabidiol (CBD) can help children with Sanfilippo syndrome, a rare genetic disorder. It may be a good fit for children who have already tried other treatments or whose disease is more advanced.
This study follows people with MPS, a rare genetic condition, to learn more about the disease over time. If you have a confirmed MPS diagnosis, you may be able to join.
This study follows people with MPS IIIC over time to learn more about how the disease progresses. It does not test a new treatment, but the information may help with future care and research.
This program provides early access to an experimental enzyme therapy given directly into the brain for children with Sanfilippo syndrome type B, a genetic condition that affects the nervous system. The treatment aims to replace a missing enzyme to slow or stabilize symptoms.
Hear when a new Sanfilippo Syndrome trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.