Sage Journals: Discover world-class research

Free access

Review article

First published 2017-7

The role of sebelipase alfa in the treatment of lysosomal acid lipase deficiency

Angelika L. Erwin

Therapeutic Advances in Gastroenterology

Open access

Review article

First published 2021

Enzyme replacement therapy in lysosomal acid lipase deficiency (LAL-D): a systematic literature review

Aamir Bashir, Pramil Tiwari, Ajay Duseja

Therapeutic Advances in Rare Disease

Retricted access

Research article

First published 2002-07

Lysosomal Acid Lipase Deficiency: Correction of Lipid Storage by Adenovirus-Mediated Gene Transfer in Mice

Hong Du, Martin Heur, David P. Witte [...]

Human Gene Therapy

Retricted access

Research article

First published 1999-11

Cholesteryl Ester Storage Disease: Case Report during Childhood

Zuhal Akçören, Safiye Göğüş, Nurten Koçak [...]

Pediatric and Developmental Pathology

Free access

Research article

First published 2019-1

Estimation of the prevalence of cholesteryl ester storage disorder in a cohort of patients with clinical features of familial hypercholesterolaemia

Pauline Ashfield-Watt, Kate Haralambos, Rhiannon Edwards [...]

Annals of Clinical Biochemistry: International Journal of Laboratory Medicine

Retricted access

Other

First published 2007-12

Cholesterol Ester Storage Disease With Unusual Neurological Manifestations in Two Siblings: A Report From South India

P.S. Bindu, A.B. Taly, R. Christopher [...]

Journal of Child Neurology

Retricted access

Research article

First published 2001-02

Phenotypic Correction of Lipid Storage and Growth Arrest in Wolman Disease Fibroblasts by Gene Transfer of Lysosomal Acid Lipase

Uwe J.F. Tietge, Gwoshing Sun, Susan Czarnecki [...]

Human Gene Therapy

Retricted access

Research article

First published 2011-06

Wolman Disease (LIPA p.G87V) Genotype Frequency in People of Iranian-Jewish Ancestry

Yadira Valles-Ayoub, Saghi Esfandiarifard, Daniel No [...]

Genetic Testing and Molecular Biomarkers

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Research article

First published 2023-10

Prevalence of p.G87V and p.Gln298=Variations in LIPA Gene Within Middle Eastern Population Living Around Los Angeles

Jayden Jackson, Justin Farajzadeh, Robert Turner [...]

Genetic Testing and Molecular Biomarkers

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