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Pediatric Neuron Disease Gets Its First Gene Therapy

Source link : https://www.newshealth.biz/health-news/pediatric-neuron-disease-gets-its-first-gene-therapy/

The FDA approved atidarsagene autotemcel (arsa-cel; Lenmeldy), the first gene therapy to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD), the agency announced on Monday. “This is the first FDA-approved treatment option for children who have this rare genetic disease,” Peter Marks, MD, PhD, director of […]

Author : News Health

Publish date : 2024-03-18 17:28:34

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