Wolman disease

Common Name(s)

Wolman disease, Lysosomal acid lipase deficiency

Wolman disease is a type of lysosomal storage disorder. It is an inherited condition that causes a buildup of lipids (fats) in body organs and calcium deposits in the adrenal glands. Common symptoms in infants include enlarged liver and spleen, poor weight gain, low muscle tone, jaundice, vomiting, diarrhea, developmental delay, anemia, and poor absorption of nutrients from food. The condition is severe and life-threatening, however new therapies, such as hematopoietic cell transplantation, have shown promise in improving the outlook of children with this disease.
 

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Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Wolman disease" for support, advocacy or research.

There are currently no organizations listed in Disease InfoSearch that support this condition. Create a listing.

 

 

General Support Organizations

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Scientific Literature

Articles from the PubMed Database

Research articles describe the outcome of a single study. They are the published results of original research.
The terms "Wolman disease" returned 17 free, full-text research articles on human participants. First 3 results:

Neural stem cells for disease modeling of Wolman disease and evaluation of therapeutics.
 

Author(s): Francis Aguisanda, Charles D Yeh, Catherine Z Chen, Rong Li, Jeanette Beers, Jizhong Zou, Natasha Thorne, Wei Zheng

Journal:

 

Wolman disease (WD) is a rare lysosomal storage disorder that is caused by mutations in the LIPA gene encoding lysosomal acid lipase (LAL). Deficiency in LAL function causes accumulation of cholesteryl esters and triglycerides in lysosomes. Fatality usually occurs within the first ...

Last Updated: 31 Dec 1969

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Morphology of Wolman cholesteryl ester storage disease.
 

Author(s): Robin M Ireland

Journal: Blood. 2017 02;129(6):803.

 

Last Updated: 31 Dec 1969

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Wolman disease in an infant.
 

Author(s): Poonam Shenoy, Lakshmikanth Karegowda, Smiti Sripathi, Nahas Mohammed

Journal:

 

Last Updated: 31 Dec 1969

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Reviews from the PubMed Database

Review articles summarize what is currently known about a disease. They discuss research previously published by others.
The terms "Wolman disease" returned 0 free, full-text review articles on human participants.

 
 
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Clinical Trial Information This information is provided by ClinicalTrials.gov

Biomarker for Wolman Disease
 

Status: Recruiting

Condition Summary: Wolman Disease; Acid Lipase Deficiency; Acid Cholesteryl Ester Hydrolase Deficiency, Wolman Type; Cholesterol Ester Storage Disease

 

Last Updated: 3 Jul 2017

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Lysosomal Acid Lipase (LAL) Deficiency Registry
 

Status: Recruiting

Condition Summary: Lysosomal Acid Lipase Deficiency; Cholesterol Ester Storage Disease; Wolman Disease; Acid Cholesteryl Ester Hydrolase Deficiency, Type 2; Acid Lipase Deficiency; LIPA Deficiency; LAL-Deficiency

 

Last Updated: 1 Mar 2018

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Lysosomal Acid Lipase Increase After Chetogenic Diet.
 

Status: Recruiting

Condition Summary: Lysosomal Acid Lipase Deficiency; Obesity; Ketogenic Dieting; Ketosis

 

Last Updated: 20 Jun 2018

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