Gene therapy for aspartylglucosaminuria
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a gene therapy given by infusion that aims to replace the faulty gene causing aspartylglucosaminuria (AGU), a rare inherited condition affecting the brain and body. The goal is to see if it's safe and can slow or improve symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed genetic diagnosis of AGU (with two changes in the AGA gene).
- You must already be enrolled in the AGU Natural History Study.
- You must be willing and able to travel to the study site and follow all study procedures.
- You must not have had a serious illness needing hospitalization in the past 6 weeks.
- You must not need breathing support (like a ventilator) on a regular basis.
- You must not have a bleeding disorder or take medicines that increase bleeding risk.
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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