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

Wolman disease

Registry of Patients Diagnosed With Lysosomal Storage Diseases Prevalence and Risk Factors of Women Mental Health Disorders Breakfast for Young Women In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102 Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency Lysosomal Acid Lipase (LAL) Deficiency Registry