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Clinical Trials for Lysosomal Storage Diseases

• Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
• A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
• Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I
• Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
• Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I