Recommendations for clinical monitoring of patients with acid sphingomyelinase deficiency (ASMD)-Reference-Cited by-同舟云学术

Recommendations for clinical monitoring of patients with acid sphingomyelinase deficiency (ASMD)

Published:2019-02 Issue:2 Volume:126 Page:98-105
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Wasserstein Melissa,Dionisi-Vici Carlo^ORCID,Giugliani Roberto^ORCID,Hwu Wuh-Liang,Lidove Olivier,Lukacs Zoltan,Mengel Eugen,Mistry Pramod K.,Schuchman Edward H.,McGovern Margaret

Funder

Sanofi Genzyme

Publisher

Elsevier BV

Subject

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

Reference45 articles.

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2. Types A and B Niemann-Pick disease;Schuchman;Mol. Genet. Metab.,2017

3. Natural history of type A Niemann-Pick disease: possible endpoints for therapeutic trials;McGovern;Neurology,2006

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

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

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