Enzyme replacement therapy for children with acid sphingomyelinase deficiency in the real world: A single center experience in Taiwan-Reference-Cited by-同舟云学术

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Enzyme replacement therapy for children with acid sphingomyelinase deficiency in the real world: A single center experience in Taiwan

Published:2023-03 Issue: Volume:34 Page:100957
ISSN:2214-4269
Container-title:Molecular Genetics and Metabolism Reports
language:en
Short-container-title:Molecular Genetics and Metabolism Reports

Author:

Pan Yu-Wen,Tsai Meng-Che,Yang Chiao-Yu,Yu Wen-Hao,Wang Bow,Yang Yao-Jong,Chou Yen-Yin

Funder

National Cheng Kung University Hospital

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology

Reference44 articles.

1. Consensus recommendation for a diagnostic guideline for acid sphingomyelinase deficiency;McGovern;Genet. Med.,2017

2. Recommendations for clinical monitoring of patients with acid sphingomyelinase deficiency (ASMD);Wasserstein;Mol. Genet. Metab.,2019

3. Disease manifestations and burden of illness in patients with acid sphingomyelinase deficiency (ASMD);McGovern;Orphanet J. Rare Dis.,2017

4. Epidemiology and diagnosis of lysosomal storage disorders; challenges of screening;Kingma;Best Pract. Res. Clin. Endocrinol. Metab.,2015

5. Cause of death in patients with chronic visceral and chronic neurovisceral acid sphingomyelinase deficiency (Niemann-pick disease type B and B variant): literature review and report of new cases;Cassiman;Mol. Genet. Metab.,2016

Cited by 6 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. Desensitization of olipudase alfa-induced anaphylaxis in a child with chronic neurovisceral acid sphingomyelinase deficiency;Molecular Genetics and Metabolism Reports;2024-09

2. Niemann-Pick Disease, Type A: Clinical Case of 5 Months Old Patient;Current Pediatrics;2024-07-12

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

4. Overview of clinical, molecular, and therapeutic features of Niemann–Pick disease (types A, B, and C): Focus on therapeutic approaches;Cell Biochemistry and Function;2024-05-07

5. Olipudase Alfa in Non-CNS Manifestations of Acid Sphingomyelinase Deficiency: A Profile of Its Use;Clinical Drug Investigation;2023-05

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