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Lysosomal Acid Lipase DeficiencyRare disease

What this study is

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.

Study details

Official title
An Observational Disease and Clinical Outcomes Registry of Patients With Lysosomal Acid Lipase (LAL) Deficiency
Sponsor
Alexion Pharmaceuticals, Inc.
Conditions
Lysosomal Acid Lipase Deficiency · Cholesterol Ester Storage Disease · Wolman Disease · Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 · Acid Lipase Deficiency · LIPA Deficiency · LAL-Deficiency
Enrolling
300 people
Starts
2013-05-30
Est. completion
2029-08-30
Record
View on ClinicalTrials.gov ↗