Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy-Reference-Cited by-同舟云学术

Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy

Published:2020-09 Issue:1-2 Volume:131 Page:116-123
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Jones Simon A.,McGovern Margaret,Lidove Olivier,Giugliani Roberto,Mistry Pramod K.,Dionisi-Vici Carlo,Munoz-Rojas Maria-Veronica,Nalysnyk Lubomyra,Schecter Alison D.,Wasserstein Melissa

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference63 articles.

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5. Acid sphingomyelinase deficiency: prevalence and characterization of an intermediate phenotype of Niemann-Pick disease;Wasserstein;J. Pediatr.,2006

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1. Natural disease course of chronic visceral acid sphingomyelinase deficiency in adults: A first step toward treatment criteria;Journal of Inherited Metabolic Disease;2024-08-23

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3. Acid sphingomyelinase deficiency and Gaucher disease in adults: Similarities and differences in two macrophage storage disorders;JIMD Reports;2024-07-04

4. Lung Diseases and Rare Disorders: Is It a Lysosomal Storage Disease? Differential Diagnosis, Pathogenetic Mechanisms and Management;Children;2024-05-30

5. Assessment of health state utilities associated with adult and pediatric acid sphingomyelinase deficiency (ASMD);The European Journal of Health Economics;2024-02-27