Improving newborn screening test performance for metachromatic leukodystrophy: Recommendation from a pre-pilot study that identified a late-infantile case for treatment-Reference-Cited by-同舟云学术

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Improving newborn screening test performance for metachromatic leukodystrophy: Recommendation from a pre-pilot study that identified a late-infantile case for treatment

Published:2024-05 Issue:1 Volume:142 Page:108349
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

WU Teresa H.Y.,Brown Heather A.,Church Heather J.,Kershaw Christopher J.,Hutton Rebekah,Egerton Christine,Cooper James,Tylee Karen,Cohen Rebecca N.,Gokhale David,Ram Dipak,Morton Georgina,Henderson Michael,Bigger Brian W.,Jones Simon A.

Funder

Orchard Therapeutics

Publisher

Elsevier BV

Reference34 articles.

1. Metachromatic Leukodystrophy;von Figura,2001

2. Association of age at onset and first symptoms with disease progression in patients with metachromatic Leukodystrophy;Kehrer;Neurology.,2021

3. Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic Leukodystrophy;Martin;Biol. Blood Marrow Transplant.,2013

4. Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies;van den Broek;Blood Adv.,2018

5. Atidarsagene Autotemcel for Treating Metachromatic Leukodystrophy. NICE Guidance;National Institute for Health and Care Excellence,2022

Cited by 1 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Current Status of Newborn Bloodspot Screening Worldwide 2024: A Comprehensive Review of Recent Activities (2020–2023);International Journal of Neonatal Screening;2024-05-23

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