Ignite Creation Date:
2025-12-25 @ 12:52 AM
Ignite Modification Date:
2026-02-12 @ 1:44 AM
Study NCT ID:
NCT02193867
Status:
TERMINATED
Last Update Posted:
2019-11-18
First Post:
2014-07-07
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
Sponsor:
Alexion Pharmaceuticals, Inc.
Organization:
Conditions & Keywords Data
Conditions:
Conditions
| Condition Brief | Condition Text | View |
|---|---|---|
| None | Lysosomal Acid Lipase Deficiency | View |
Keywords:
Keywords
| Keyword Brief | Keyword Text | View |
|---|---|---|
| None | LIPA | View |
| None | Wolman Disease | View |
| None | Wolman Phenotype | View |
| None | Acid Lipase Deficiency | View |
| None | Acid Cholesteryl Hydrolase | View |
| None | Acid Lipase Disease Deficiency, type 2 | View |
| None | Cholesteryl Ester Storage Disease (CESD) | View |
| None | Cholesteryl Ester Hydrolase Deficiency | View |
| None | Early Onset Lysosomal Acid Lipase Deficiency (Wolman Disease) | View |
| None | LAL Deficiency | View |
| None | Late Onset Lysosomal Acid Lipase Deficiency (CESD) | View |
| None | Wolman Disease (early onset LAL Deficiency) | View |
| None | Related Disorders: | View |
| None | Non-alcoholic Fatty Liver Disease (NAFLD) | View |
| None | Non-alcoholic Steatohepatitis (NASH) | View |
| None | Alcoholic Liver Disease | View |
| None | Cryptogenic Cirrhosis | View |
| None | Niemann-Pick Disease (NPD) Type C | View |
| None | Chanarin Dorfman Syndrome | View |