| Enrolling By Invitation | Long Term Follow-Up for RGX-111 NCT06103487 | REGENXBIO Inc. | — |
| Completed | Baby Detect : Genomic Newborn Screening NCT05687474 | Centre Hospitalier Universitaire de Liege | — |
| Recruiting | Registry of Patients Diagnosed With Lysosomal Storage Diseases NCT05619900 | University of California, San Francisco | — |
| Completed | An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I) NCT04453085 | JCR Pharmaceuticals Co., Ltd. | Phase 1 / Phase 2 |
| Completed | China Post-marketing Surveillance (PMS) Study of Aldurazyme® NCT05134571 | Genzyme, a Sanofi Company | Phase 4 |
| Active Not Recruiting | Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX NCT04628871 | Sangamo Therapeutics | — |
| Completed | A Study of JR-171 in Patients With Mucopolysaccharidosis I NCT04227600 | JCR Pharmaceuticals Co., Ltd. | Phase 1 / Phase 2 |
| Recruiting | MPS (RaDiCo Cohort) (RaDiCo-MPS) NCT06036693 | Institut National de la Santé Et de la Recherche Médicale, France | — |
| Active Not Recruiting | Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI NCT03153319 | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | Phase 1 / Phase 2 |
| Completed | Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I NCT03071341 | ArmaGen, Inc | Phase 1 / Phase 2 |
| Completed | Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181 NCT02597114 | ArmaGen, Inc | Phase 1 |
| Completed | Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MP NCT03053089 | ArmaGen, Inc | Phase 1 / Phase 2 |
| Completed | Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I NCT02371226 | ArmaGen, Inc | Phase 1 |
| Terminated | Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I NCT02232477 | agnes chen | N/A |
| Completed | Human Placental-Derived Stem Cell Transplantation NCT01586455 | New York Medical College | Phase 1 |
| Terminated | Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplas NCT01675674 | National MPS Society | — |
| Terminated | A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysacch NCT00418821 | Genzyme, a Sanofi Company | Phase 4 |
| Completed | A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I NCT00852358 | Patricia I. Dickson, M.D. | N/A |
| Terminated | Clinical Trial of Growth Hormone in MPS I, II, and VI NCT00748969 | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | Phase 2 / Phase 3 |
| Completed | Immune Tolerance Study With Aldurazyme® (Laronidase) NCT00741338 | Genzyme, a Sanofi Company | Phase 1 / Phase 2 |
| Terminated | Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I NCT00786968 | Patricia I. Dickson, M.D. | Phase 1 |
| Completed | Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products NCT00695279 | St. Jude Children's Research Hospital | — |
| Completed | Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease NCT00258011 | Genzyme, a Sanofi Company | Phase 3 |
| Terminated | Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I NCT00215527 | Patricia I. Dickson, M.D. | Phase 1 |
| Completed | A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease NCT00144781 | Genzyme, a Sanofi Company | Phase 4 |
| Completed | A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosa NCT00144768 | Genzyme, a Sanofi Company | Phase 4 |
| Completed | Stem Cell Transplant w/Laronidase for Hurler NCT00176891 | Masonic Cancer Center, University of Minnesota | Phase 2 |
| Completed | A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Y NCT00146757 | Genzyme, a Sanofi Company | Phase 2 |
| Completed | Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS NCT00146770 | Genzyme, a Sanofi Company | Phase 3 |
| Completed | Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I NCT00912925 | Genzyme, a Sanofi Company | Phase 3 |
| Unknown | Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoie NCT00005900 | Fairview University Medical Center | — |
| Completed | Stem Cell Transplantation for Hurler NCT00176917 | Masonic Cancer Center, University of Minnesota | Phase 2 |