Survival of patients with chronic acid sphingomyelinase deficiency (ASMD) in the United States: A retrospective chart review study-Reference-Cited by-同舟云学术

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Survival of patients with chronic acid sphingomyelinase deficiency (ASMD) in the United States: A retrospective chart review study

Published:2024-03 Issue: Volume:38 Page:101040
ISSN:2214-4269
Container-title:Molecular Genetics and Metabolism Reports
language:en
Short-container-title:Molecular Genetics and Metabolism Reports

Author:

Pulikottil-Jacob Ruth,Dehipawala Sumudu,Smith Brittany,Athavale Amod,Gusto Gaelle,Chandak Aastha,Khachatryan Artak,Banon Tamar,Fournier Marie,Guillonneau Sophie,Pollissard Laurence,Munoz-Rojas Maria Veronica

Funder

Sanofi

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology

Reference23 articles.

1. A prospective, cross-sectional survey study of the natural history of Niemann-Pick disease type B;McGovern;Pediatrics.,2008

2. Types A and B Niemann-Pick disease;Schuchman;Best Pract. Res. Clin. Endocrinol. Metab.,2015

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

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

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

Cited by 1 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

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