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Clinical Trials for Wolman Disease

• In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases
• An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
• Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
• Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
• Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
• Human Placental-Derived Stem Cell Transplantation
• Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
• Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
• Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
• Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency
• Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
• HSCT for High Risk Inherited Inborn Errors
• Stem Cell Transplant for Inborn Errors of Metabolism