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Lysosomal Acid Lipase (LAL) Deficiency Registry

Lysosomal Acid Lipase Deficiency | Wolman Disease | Acid Lipase Deficiency | Cholesterol Ester Storage...

This is an observational, multi-center, international disease registry designed to collect longitudinal data and create a knowledge base that will be utilized to improve the care and treatment of patients with LAL Deficiency. Participation in the Registry by both physicians and patients is voluntary.

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Participation Requirements

  • Sex:

    ALL
  • Eligible Ages:

    0 and up

Participation Criteria

Patients must have a confirmed diagnosis of LAL Deficiency. An Informed Consent and Authorization must be obtained prior to patient enrollment where required under applicable laws and regulations, or a waiver must be obtained by the Institutional Review Board/Independent Ethics Committee.

Patients cannot be currently participating in an Alexion-sponsored clinical trial. Patients who have concluded participation in an Alexion-sponsored sebelipase alfa clinical trial are eligible to enroll in this Registry, and enrollment in the Registry will not exclude a patient from enrolling in a future clinical trial.

Study Location


London, Ontario
Canada

Contact Study Team

Study Sponsored By
Alexion Pharmaceuticals, Inc.
Participants Required
More Information
Study ID: NCT01633489