Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

Mucopolysaccharidosis type I

Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH. China Post-marketing Surveillance (PMS) Study of Aldurazyme® The Intensively Follow-up Examinations for Asymptomatic MPS I Infants in Taiwan Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers An Extension Study of JR-171-101 Study in Patients With MPS I Stem Cell Transplant w/Laronidase for Hurler Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181 A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant Biomarker for Hurler Disease (BioHurler) A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I and II Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I Immune Tolerance Study With Aldurazyme® (Laronidase) A Study of JR-171 in Patients With Mucopolysaccharidosis I A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I Mucopolysaccharidosis I (MPS I) Registry Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I RGX-111 Gene Therapy in Patients With MPS I Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I MRS to Determine Neuroinflammation and Oxidative Stress in MPS I Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease