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Clinical Trials for Mucopolysaccharidoses

• Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
• Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
• An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
• An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Male Subjects
• A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)
• A Study to the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
• A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
• China Post-marketing Surveillance (PMS) Study of Aldurazyme®
• The Effect of Enzyme Replacement Therapy in Mucopolysaccharidosis
• A Phase Ⅲ Study of JR-141 in Patients With Mucopolysaccharidosis II
• An Extension Study of JR-171-101 Study in Patients With MPS I
• A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102
• An Extension Study of JR-141 in Patients With Mucopolysaccharidosis II
• MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT
• A Study of DNL310 in Pediatric Participants With Hunter Syndrome
• A Study of JR-171 in Patients With Mucopolysaccharidosis I
• Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA
• Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
• A Treatment Extension Study of Mucopolysaccharidosis Type IIIB
• An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
• Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI
• Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
• A Study of JR-141 in Patients With Mucopolysaccharidosis II
• A Study in MPS VI to Assess Safety and Efficacy of Odiparcil
• A Study of JR-141 in Patients With Mucopolysaccharidosis II
• Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I and II
• A Study of JR-141 in Patients With Mucopolysaccharidosis Type II
• A Treatment Study of Mucopolysaccharidosis Type IIIB
• A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously
• 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
• A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
• Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
• Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I
• An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.
• Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
• A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
• Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I
• A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
• MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
• An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
• BMN 110 Phase 3B in Australian Patients
• BMN 110 US Expanded Access Program
• An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
• Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
• Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
• A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723
• Human Placental-Derived Stem Cell Transplantation
• Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
• Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
• Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
• To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
• A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
• A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
• Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis
• Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
• A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
• 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)
• Intrathecal Enzyme Replacement for Hurler Syndrome
• A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
• 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
• Study of rhASB in Patients With Mucopolysaccharidosis VI
• Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
• Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
• Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI