MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT

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 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) 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 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 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 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 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 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