An Observational Disease and Clinical Outcomes Registry of Patients With Lysosomal Acid Lipase (LAL) Deficiency
Latest Information Update: 09 Jun 2025
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