Miglustat in patients with Niemann-Pick disease Type C (NP-C): A multicenter observational retrospective cohort study-Reference-Cited by-同舟云学术

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Miglustat in patients with Niemann-Pick disease Type C (NP-C): A multicenter observational retrospective cohort study

Published:2009-11 Issue:3 Volume:98 Page:243-249
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Pineda M.,Wraith J.E.,Mengel E.,Sedel F.,Hwu W.-L.,Rohrbach M.,Bembi B.,Walterfang M.,Korenke G.C.,Marquardt T.

Publisher

Elsevier BV

Subject

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

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4. Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study;Orphanet Journal of Rare Diseases;2023-07-21

5. Current advancements in therapy for Niemann-Pick disease: progress and pitfalls;Expert Opinion on Pharmacotherapy;2023-05-21

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