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129 studies found for:    "Mucopolysaccharidosis"
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Rank Status Study
1 Unknown  Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis
Condition: Mucopolysaccharidosis
Interventions: Drug: Conditioning regimen;   Drug: Graft-versus-host disease (GVHD) prophylaxis;   Procedure: Stem Cell Transplantation
2 Completed Natural History Studies of Mucopolysaccharidosis III
Conditions: Mucopolysaccharidosis Type IIIA;   Mucopolysaccharidosis Type IIIB
Interventions: Procedure: Lumbar puncture;   Device: Magnetic Resonance Imaging (MRI) of the brain
3 Completed
Has Results
Study of rhASB in Patients With Mucopolysaccharidosis VI
Condition: Mucopolysaccharidosis VI
Interventions: Drug: N-acetylgalactosamine 4-sulfatase;   Drug: Placebo/rhASB
4 Recruiting Mucopolysaccharidosis I (MPS I) Registry
Condition: Mucopolysaccharidosis I (MPS I)
Intervention:
5 Not yet recruiting Mucopolysaccharidosis (MPS) Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia Combined With Pediatric ENT Surgery or Bronchoscopy
Condition: Mucopolysaccharidoses
Intervention:
6 Terminated Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
Condition: Mucopolysaccharidosis Type IVA
Intervention:
7 Completed Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
Conditions: Mucopolysaccharidosis IV A;   Mucopolysaccharidosis VI
Intervention: Other: Enzyme testing
8 Completed
Has Results
Immune Tolerance Study With Aldurazyme® (Laronidase)
Condition: Mucopolysaccharidosis I
Interventions: Biological: Laronidase;   Drug: Cyclosporine A (CsA);   Drug: Azathioprine (Aza)
9 Unknown  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 MPS 7
Condition: Mucopolysaccharidosis Type 7
Intervention: Drug: UX003
10 Completed Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)
Condition: Mucopolysaccharidosis II
Intervention: Genetic: lymphocyte gene therapy
11 Recruiting Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI
Conditions: Mucopolysaccharidosis Type I;   Mucopolysaccharidosis Type II;   Mucopolysaccharidosis Type VI
Interventions: Drug: Adalimumab;   Other: Placebo
12 Recruiting A Treatment Study of Mucopolysaccharidosis Type IIIB
Conditions: MPS III B;   Mucopolysaccharidosis Type IIIB
Intervention: Drug: BMN 250
13 Recruiting Longitudinal Study of Bone Disease in Children With Mucopolysaccharidoses (MPS) I, II, and VI
Condition: Mucopolysaccharidoses
Intervention:
14 Completed
Has Results
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
Condition: Mucopolysaccharidosis II
Interventions: Biological: Iduronate-2-sulfatase enzyme replacement therapy;   Biological: iduronate-2-sulfatase enzyme replacement therapy;   Biological: Placebo
15 Recruiting Natural History Study of Patients With MPS IIIA
Condition: Mucopolysaccharidosis IIIA
Intervention:
16 No longer available An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Condition: Mucopolysaccharidosis Type 7
Intervention: Drug: UX003
17 Active, not recruiting Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I
Condition: Mucopolysaccharidosis I
Intervention: Drug: AGT-181
18 Not yet recruiting Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
Condition: Mucopolysaccharidosis II
Intervention: Biological: SB-913
19 Active, not recruiting Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I
Condition: Mucopolysaccharidosis I
Intervention: Drug: AGT-181 (HIRMAb-IDUA)
20 Completed To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
Condition: Mucopolysaccharidosis II
Interventions: Drug: GC1111_0.5mg/kg;   Drug: GC1111_1.0mg/kg;   Drug: Elaprase_0.5mg/kg

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Study has passed its completion date and status has not been verified in more than two years.