Cerliponase alfa changes the natural history of children with neuronal ceroid lipofuscinosis type 2: The first French cohort
Author:
Publisher
Elsevier BV
Subject
Neurology (clinical),General Medicine,Pediatrics, Perinatology and Child Health
Reference23 articles.
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2. Clinical, electrophysiological, imaging, and ultrastructural description in 68 patients with neuronal ceroid lipofuscinoses and its subtypes;Jadav;Pediatric Neurol.,2014
3. Neuronal ceroid lipofuscinoses;Chabrol;Handb. Clin. Neurol.,2013
4. NCL diseases — clinical perspectives;Schulz;Biochim. Biophys. Acta (BBA) - Mol. Basis Dis.;nov,2013
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1. Ceroid lipofuscinosis type 2 disease: Effective presymptomatic therapy—Oldest case of a presymptomatic enzyme therapy;European Journal of Neurology;2024-05
2. Classic and Atypical Late Infantile Neuronal Ceroid Lipofuscinosis in Latin America: Clinical and Genetic Aspects, and Treatment Outcome with Cerliponase Alfa;Molecular Genetics and Metabolism Reports;2024-03
3. Case Report: Intraventricular Cerliponase Alfa Treatment in a Patient with Advanced Neuronal Ceroid Lipofuscinosis Type 2;Internal Medicine;2024
4. Clinical management and diagnosis of CLN2 disease: consensus of the Brazilian experts group;Arquivos de Neuro-Psiquiatria;2023-03
5. Cerliponase alfa in the treatment of patients with classic and atypical late infantile neuronal ceroid lipofuscinosis in Latin America;2022-11-28
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