Sebelipase alfa for lysosomal acid lipase deficiency: 5‐year treatment experience from a phase 2 open‐label extension study-Reference-Cited by-同舟云学术

Sebelipase alfa for lysosomal acid lipase deficiency: 5‐year treatment experience from a phase 2 open‐label extension study

Published:2020-08-09 Issue:9 Volume:40 Page:2203-2214
ISSN:1478-3223
Container-title:Liver International
language:en
Short-container-title:Liver Int

Author:

Malinová Vĕra¹^ORCID,Balwani Manisha²,Sharma Reena³,Arnoux Jean‐Baptiste⁴,Kane John⁵,Whitley Chester B.⁶,Marulkar Sachin⁷,Abel Florian⁷

Affiliation:

1. Department for Metabolic Diseases Children's ClinicGeneral Faculty Hospital and First Faculty of Medicine of Charles University in Prague Prague Czech Republic

2. Department of Genetics and Genomic Sciences and Department of Medicine Icahn School of Medicine at Mount Sinai New York NY USA

3. Department of Endocrinology and Metabolic Medicine Salford Royal Foundation NHS Trust Salford UK

4. Department of Inherited Metabolic Diseases Hôpital Necker‐Enfants Malades Paris France

5. Department of Medicine University of California San Francisco San Francisco CA USA

6. Advanced Therapies Program, and Gene Therapy Center University of Minnesota Minneapolis MN USA

7. Alexion Pharmaceuticals, Inc. Boston MA USA

Publisher

Wiley

Subject

Hepatology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/liv.14603

Reference18 articles.

1. Lysosomal Acid Lipase Deficiency — A New Therapy for a Genetic Lipid Disease

2. Lysosomal acid lipase deficiency – An under-recognized cause of dyslipidaemia and liver dysfunction

3. Cholesteryl ester storage disease: Review of the findings in 135 reported patients with an underdiagnosed disease

4. A new method for the measurement of lysosomal acid lipase in dried blood spots using the inhibitor Lalistat 2

Cited by 17 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. Otras enfermedades hepáticas de causa genética, metabólica y endocrinológica;Medicine - Programa de Formación Médica Continuada Acreditado;2024-03

2. Lanifibranor Reduces Inflammation and Improves Dyslipidemia in Lysosomal Acid Lipase-Deficient Mice;Gastro Hep Advances;2024

3. Clinical guidelines for the management of children with lysosomal acid lipase deficiency;Pediatric pharmacology;2023-08-22

4. Recent insights into lysosomal acid lipase deficiency;Trends in Molecular Medicine;2023-06

5. Lysosomal acid lipase deficiency manifestations in children and adults: Baseline data from an international registry;Liver International;2023-05-24