Gene Therapy Shows Long-Term Benefits in Pediatric Neuron Disease

Watchdoq April 24, 2025
(MedPage Today) -- Severe motor impairment or death was significantly less common than typical among children with late-infantile or early-juvenile metachromatic leukodystrophy (MLD) after they received atidarsagene autotemcel (arsa-cel; Lenmeldy...

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