Metachromatic leukodystrophy is a rare genetic disorder that mainly affects young children and results in severe neurological symptoms accompanied by a loss of motor and intellectual capacities.
New gene therapy for metachromatic leukodystrophy proves effective in mice retrieved 22 April 2024 from https://medicalxpress.com/news/2024-04-gene-therapy-metachromatic-leukodystrophy-effective.html
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