Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Inclusion Criteria: * Participant's parent or legal guardian provided written informed consent/permission prior to any study procedures. * Male or female child with documented decreased LAL activity relative to the normal range of the laboratory performing the assay or documented result of molecular genetic testing (2 mutations) confirming a diagnosis. * Growth failure with onset before 6 months of age. Exclusion Criteria: * Clinically important concurrent disease or comorbidities. * Had received an investigational product other than sebelipase alfa within 14 days prior to the first dose. * Participant was older than 24 months of age. * Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplant. * Previous hematopoietic stem cell or liver transplant. * Known hypersensitivity to eggs.