Nils Janzen

Clinical management of metachromatic leukodystrophy cases identified in the German and Austrian Newborn screening pilots

Abstract Background Metachromatic leukodystrophy (MLD) is a rapidly progressive leukodystrophy that leads to severe disability and early death if untreated. Autologous hematopoietic stem and progenitor cell gene therapy (HSPC-GT, atidarsagene autotemcel, arsa-cel) for early onset...