NCT03632213
Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI treatment 2 active_not_recruiting NCT03150069
Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them? No drug interventions Not Available Not Available completed NCT01822184
Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II) No drug interventions Not Available Not Available completed NCT05494593
A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II) treatment 4 recruiting NCT02350816
An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. treatment 2 terminated NCT01155778
Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients treatment 1 / 2 completed NCT05155488
A Study to Improve the Awareness of Mucopolysaccharidosis Type II in Brazil No drug interventions Not Available Not Available completed NCT02095015
Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project) No drug interventions Not Available Not Available terminated NCT00654433
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases No drug interventions treatment 3 terminated NCT01695161
Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer. No drug interventions Not Available Not Available unknown_status NCT01238328
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis treatment 2 / 3 unknown_status NCT02418455
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 treatment 2 completed NCT05006222
The Effect of Enzyme Replacement Therapy in Mucopolysaccharidosis No drug interventions supportive_care Not Available completed NCT02230566
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) treatment 3 completed NCT02432144
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) treatment 3 completed NCT01043640
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders treatment 2 completed