One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency-Reference-Cited by-同舟云学术

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One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency

Published:2021-08 Issue:8 Volume:23 Page:1543-1550
ISSN:1098-3600
Container-title:Genetics in Medicine
language:en
Short-container-title:Genetics in Medicine

Author:

Diaz George A.,Jones Simon A.,Scarpa Maurizio,Mengel Karl Eugen,Giugliani Roberto,Guffon Nathalie,Batsu Isabela,Fraser Patricia A.,Li Jing,Zhang Qi,Ortemann-Renon Catherine

Publisher

Elsevier BV

Subject

Genetics (clinical)

Link

https://www.nature.com/articles/s41436-021-01156-3.pdf

Reference34 articles.

Cited by 52 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. Acid sphingomyelinase deficiency in France: a retrospective survival study;Orphanet Journal of Rare Diseases;2024-08-05

2. Natural history of acid sphingomyelinase deficiency among European patients during childhood and adolescence: A retrospective observational study;European Journal of Medical Genetics;2024-08

3. The Liver and Lysosomal Storage Diseases: From Pathophysiology to Clinical Presentation, Diagnostics, and Treatment;Diagnostics;2024-06-19

4. Maladies de Niemann-Pick ou déficits en sphingomyélinase acide (ASMD) : présentations cliniques, actualités thérapeutiques et perspectives;La Revue de Médecine Interne;2024-06

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

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