Trial Profile

An Observational Disease and Clinical Outcomes Registry of Patients With Lysosomal Acid Lipase (LAL) Deficiency

Status: Recruiting

Phase of Trial: Phase IV

Latest Information Update: 09 Jun 2025

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At a glance

Drugs Sebelipase alfa (Primary)
Indications Cholesterol ester storage disease
Focus Therapeutic Use
Sponsors Alexion AstraZeneca Rare Disease

Most Recent Events

09 Jun 2025 New trial record

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