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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
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