Lysosomal Acid Lipase (LAL) Deficiency is a rare condition where the body doesn't have enough of an enzyme called LAL, which helps break down fats. This can lead to serious health issues, including liver and heart problems. There are two main types: one affects infants and is very dangerous, while the other affects children and adults. The LAL Deficiency Registry is a global project to collect information from patients with this condition to help doctors and researchers learn more about it. The registry aims to improve treatment and care for patients. It's important to note that both doctors and patients choose to join this registry voluntarily.
- Participation requires confirmed LAL Deficiency diagnosis.
- Patients cannot be in any other Alexion-sponsored clinical trial at the same time.
- The registry helps gather data to improve care and treatment.
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