Gene therapy for MLD using the patient's own stem cells
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene therapy that uses a patient's own blood stem cells to treat MLD (metachromatic leukodystrophy), a rare genetic disorder that damages the nervous system. The goal is to see if this treatment can stop or slow the disease.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You or your child must have a confirmed diagnosis of MLD through a genetic test showing a mutation in the ARSA gene.
- The patient must be at least 1 month old.
- Patients must be able to have a brain MRI scan.
- They must not have HIV or any uncontrolled infection, cancer, or serious heart, liver, or kidney problems.
- The patient must not have any other neurological condition besides MLD.
- The family must be willing to sign consent and accept the risks of the trial.
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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