Cholesterol Ester Storage Disease
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CLINICAL_TRIALS research · 2026-03-06Clinical Trial Update: Lysosomal Acid Lipase (LAL) Deficiency Registry
Clinical trial registry update describing a Lysosomal Acid Lipase (LAL) deficiency registry that is currently recruiting to collect data on …
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CLINICAL_TRIALS trial_result · 2018-12-11Clinical Trial Update: Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency [Phase 1, Phase 2]
Phase 1/2 trial update evaluating safety, tolerability, and pharmacokinetics of SBC-102 (Sebelipase alfa) in adults with lysosomal acid lipa…
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CLINICAL_TRIALS research · 2016-07-20Clinical Trial Update: An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype
An observational study of patients with lysosomal acid lipase deficiency and the cholesteryl ester storage disease phenotype has completed.
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CLINICAL_TRIALS trial_result · 2016-05-09Clinical Trial Update: Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency [Phase 2]
Phase 2 extension study evaluating long-term safety, tolerability, and efficacy of Sebelipase alfa (SBC-102) in adults with lysosomal acid l…
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CLINICAL_TRIALS research · 2015-07-28Clinical Trial Update: Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up
A completed observational study update describing a natural history chart review and longitudinal follow-up for Wolman disease and cholester…
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CLINICAL_TRIALS research · 2013-08-13Clinical Trial Update: Identification of Undiagnosed Lysosomal Acid Lipase Deficiency [NA]
The clinical trial update notes identification of undiagnosed lysosomal acid lipase deficiency and cholesterol ester storage disease within …
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CLINICAL_TRIALS research · 2013-02-15Clinical Trial Update: Novel Association of Cholesterol Ester Storage Disease Due to Lysosomal Acid Lipase Deficiency and Non-Alcoholic Fatty Liver Disease: A Prospective Clinical Study
A prospective clinical study investigates the association between cholesterol ester storage disease due to lysosomal acid lipase deficiency …