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Clinical Trials for Mucopolysaccharidosis I

• China Post-marketing Surveillance (PMS) Study of Aldurazyme®
• An Extension Study of JR-171-101 Study in Patients With MPS I
• MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT
• A Study of JR-171 in Patients With Mucopolysaccharidosis I
• Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
• Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I and II
• Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)
• Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181
• Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI
• Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I
• Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I
• MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
• Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
• Human Placental-Derived Stem Cell Transplantation
• Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
• Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
• Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
• A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
• Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I
• Clinical Trial of Growth Hormone in MPS I, II, and VI
• Immune Tolerance Study With Aldurazyme® (Laronidase)
• Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
• Intrathecal Enzyme Replacement for Hurler Syndrome
• Effects of Growth Hormone in Chronically Ill Children
• Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
• Stem Cell Transplantation for Hurler
• Stem Cell Transplant w/Laronidase for Hurler
• A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients