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

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

Background: Acid sphingomyelinase deficiency (ASMD), historically known as Niemann–Pick disease type A, A/B, and B, is a rare lysosomal storage pathology with multisystemic clinical manifestations. The aims of this study were to estimate the survival probability in patients in the United States with...

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Bibliographic Details
Main Authors: Ruth Pulikottil-Jacob, Sumudu Dehipawala, Brittany Smith, Amod Athavale, Gaelle Gusto, Aastha Chandak, Artak Khachatryan, Tamar Banon, Marie Fournier, Sophie Guillonneau, Laurence Pollissard, Maria Veronica Munoz-Rojas
Format: Article
Language:English
Published: Elsevier 2024-03-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Acid sphingomyelinase deficiency
Survival analyses
Mortality
Niemann–Pick disease
Chronic
Chart review
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426923000861

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http://www.sciencedirect.com/science/article/pii/S2214426923000861

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