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Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 clinical trials

View clinical trials related to Acid Cholesteryl Ester Hydrolase Deficiency, Type 2.

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NCT ID: NCT01884220 Completed - Wolman Disease Clinical Trials

Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

Start date: November 2010
Phase: N/A
Study type: Observational

The purpose of this study are: to characterize and understand the natural history of disease progression in WD and CESD, and to provide historical controls for WD and CESD for developing clinical treatment trials. The hypothesis is that the variability and clinical progression in WD and CESD is large and represents a continuum of severities from a lethal infantile to near normal adults with only "fatty livers".

NCT ID: NCT01633489 Recruiting - Clinical trials for Lysosomal Acid Lipase Deficiency

Lysosomal Acid Lipase (LAL) Deficiency Registry

ALX-LALD-501
Start date: December 2012
Phase:
Study type: Observational [Patient Registry]

This is an observational, multi-center, international disease registry designed to collect longitudinal data and create a knowledge base that will be utilized to improve the care and treatment of patients with LAL Deficiency. Participation in the Registry by both physicians and patients is voluntary.