ReviewTherapeutic advances in gastroenterology2017
The role of sebelipase alfa in the treatment of lysosomal acid lipase deficiency.
Review in Therapeutic advances in gastroenterology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
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Who cites it
10 citing papers in PubMed, 28 citations in OpenAlex.
- 15-Year progression to liver cancer in the lack of treatment for lysosomal acid lipase deficiency: A case report.Medicine · 2022Article
- Lysosomal acid lipase deficiency: A rare inherited dyslipidemia but potential ubiquitous factor in the development of atherosclerosis and fatty liver disease.Frontiers in genetics · 2022Review
- Wolman's disease presenting with secondary hemophagocytic lymphohistiocytosis: a case report from Saudi Arabia and literature review.BMC pediatrics · 2021Review
- Lysosome (Dys)function in Atherosclerosis-A Big Weight on the Shoulders of a Small Organelle.Frontiers in cell and developmental biology · 2021Review
- Cholesteryl ester storage disease of clinical and genetic characterisation: A case report and review of literature.World journal of clinical cases · 2020Article
- Inborn errors of metabolism in the differential diagnosis of fatty liver disease.The Turkish journal of gastroenterology : the official journal of Turkish Society of Gastroenterology · 2020Review
- A kinetic assay of total lipase activity for detecting lysosomal acid lipase deficiency (LAL-D) and the molecular characterization of 18 LAL-D patients from Russia.JIMD reports · 2019Article
- Hepatocyte-specific deletion of lysosomal acid lipase leads to cholesteryl ester but not triglyceride or retinyl ester accumulation.The Journal of biological chemistry · 2019Article
- Genetic and Epigenetic Culprits in the Pathogenesis of Nonalcoholic Fatty Liver Disease.Journal of clinical and experimental hepatology · 2018Review
- [Lysosomal acid lipase deficiency (LAL-D) : Diagnostic and therapeutic options in an underdiagnosed disease].Der Pathologe · 2018Review
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Authors and funding
1 author at 1 institution in 1 country.
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Abstract
Lysosomal acid lipase deficiency (LALD) is a lysosomal storage disorder (LSD) characterized either by infantile onset with fulminant clinical course and very poor prognosis or childhood/adult-onset disease with an attenuated phenotype. The disorder is often misdiagnosed or remains undiagnosed in children and adults due to a rather unspecific clinical presentation with dyslipidemia and steatohepatitis. Until recently, no good treatment options were available for LALD. Despite supportive and symptomatic therapies, death occurred before 1 year of age in patients with infantile-onset disease and patients with childhood/adult-onset LALD suffered from significant complications, such as liver cirrhosis, requiring liver transplantation and early-onset cardiovascular disease. With the recent approval of sebelipase alfa for clinical use in infantile- as well as childhood/adult-onset LALD, a new treatment era for this disorder has begun. Sebelipase alfa is a recombinant human lysosomal acid lipase (LAL), which is administered
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