Gene Therapy for Aspartylglucosaminuria (AGU)
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene therapy designed to treat aspartylglucosaminuria (AGU), a rare genetic disorder that affects how the body breaks down certain proteins. The therapy delivers a working copy of the AGA gene directly into cells to help the body function better.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed genetic diagnosis of AGU with two disease-causing mutations in the AGA gene
- You must be enrolled in the AGU Natural History Study
- You must be able to travel to the study site and attend all visits as scheduled
- You cannot have had recent hospitalization (within 6 weeks) or acute illness
- You cannot require breathing support (ventilator or oxygen assistance) on a regular basis
- You cannot have had any previous gene therapy, cell therapy, or bone marrow transplant
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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