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This is an open-label, multicenter expanded access protocol to allow patients with a confirmed diagnosis of Lysosomal Acid Lipase (LAL) Deficiency in the United States (US), access to sebelipase alfa (recombinant lysosomal acid lipase [rhLAL]) until commercial product is available.
Patients enrolled in the expanded access protocol will receive 1 mg/kg intravenous infusions of sebelipase alfa every other week.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| sebelipase alfa | Drug |
|
Inclusion Criteria:
Exclusion Criteria:
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| ID | Term |
|---|---|
| D015223 | Wolman Disease |
| D015217 | Cholesterol Ester Storage Disease |
| D016464 | Lysosomal Storage Diseases |
| ID | Term |
|---|---|
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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| ID | Term |
|---|---|
| C000603932 | Sebelipase alfa |
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| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |