Gene therapy for SLC13A5 citrate transporter disorder
Part of Genetic & congenital clinical trials.
This trial tests a new gene therapy (TSHA-105) given through a spinal tap to treat SLC13A5 citrate transporter disorder, a genetic condition that affects the brain and body. The goal is to see if it can help improve symptoms by delivering a working copy of the gene.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of SLC13A5 citrate transporter disorder through a genetic test.
- You must be between 2 and 20 years old.
- You must be able to safely have a spinal tap (lumbar puncture).
- You cannot be taking part in any other clinical trial.
- You must not have any medical condition or allergy that prevents using the study drug or the immune-suppressing medicines.
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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