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Niemann-Pick type C disease as proof-of-concept for intelligent biomarker panel selection in neurometabolic disorders. (2022)

First Author: Papandreou A

Attributed to: Development of rAAV-mediated gene therapy for a severe paediatric metabolic liver disease: Ornithine Transcarbamylase deficiency. funded by MRC

Abstract

No abstract provided

Bibliographic Information

Digital Object Identifier: http://dx.doi.org/10.1111/dmcn.15334

PubMed Identifier: 35833379

Publication URI: http://europepmc.org/abstract/MED/35833379

Type: Journal Article/Review

Volume: 64

Parent Publication: Developmental medicine and child neurology

Issue: 12

ISSN: 0012-1622