Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study-Reference-Cited by-同舟云学术

Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study

Published:2024-01 Issue:1 Volume:23 Page:60-70
ISSN:1474-4422
Container-title:The Lancet Neurology
language:en
Short-container-title:The Lancet Neurology

Author:

Schulz Angela,Specchio Nicola,de los Reyes Emily,Gissen Paul,Nickel Miriam,Trivisano Marina,Aylward Shawn C,Chakrapani Anupam,Schwering Christoph,Wibbeler Eva,Westermann Lena Marie,Ballon Douglas J,Dyke Jonathan P,Cherukuri Anu,Bondade Shailesh,Slasor Peter,Cohen Pfeffer Jessica

Funder

BioMarin Pharmaceutical

Publisher

Elsevier BV

Subject

Neurology (clinical)

Reference26 articles.

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3. Diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease): expert recommendations for early detection and laboratory diagnosis;Fietz;Mol Genet Metab,2016

4. Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study;Nickel;Lancet Child Adolesc Health,2018

5. CLN2 disease (classic late infantile neuronal ceroid lipofuscinosis);Kohlschütter;Pediatr Endocrinol Rev,2016

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1. High Prevalence of Movement Disorder in Treated CLN2-Batten Disease;Neurology;2024-08-13

2. Phenotypic/Genotypic Profile of Children with Neuronal Ceroid Lipofuscinosis in Southern Brazil;Neuropediatrics;2024-06-10

3. GABAergic interneurons contribute to the fatal seizure phenotype of CLN2 disease mice;2024-03-30

4. Cerliponase alfa and neuronal ceroid lipofuscinosis type 2: long-term outcomes and lessons for future research;The Lancet Neurology;2024-01