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127 studies found for:    Mucopolysaccharidoses

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1 Terminated Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics
Conditions: Mucopolysaccharidoses;   Mucopolysaccharidosis I;   Mucopolysaccharidosis II;   Mucopolysaccharidosis IV;   Mucopolysaccharidosis VI
Intervention: Other: Dried blood spot test for MPS
2 Terminated Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
Condition: Mucopolysaccharidosis Type IVA
Intervention:
3 Terminated 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)
Conditions: Mucopolysaccharidosis;   Mucopolysaccharidosis I;   Mucopolysaccharidosis II;   Mucopolysaccharidosis VI
Intervention:
4 Unknown † Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis
Condition: Mucopolysaccharidosis
Interventions: Drug: Conditioning regimen;   Drug: Graft-versus-host disease (GVHD) prophylaxis;   Procedure: Stem Cell Transplantation
5 Terminated Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
Conditions: Mucopolysaccharidosis I;   Lysosomal Storage Diseases;   Spinal Cord Compression
Intervention: Drug: laronidase
6 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
7 Active, not recruiting Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Conditions: Mucopolysaccharidosis IV A;   Morquio A Syndrome;   MPS IVA
Interventions: Drug: BMN 110 - Weekly;   Drug: BMN 110 - Every Other Week
8 Terminated
Has Results 		A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Conditions: MPS IV A;   Mucopolysaccharidosis IVA;   Morquio A Syndrome
Intervention: Drug: BMN 110
9 Terminated A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Conditions: MPS IV A;   Mucopolysaccharidosis IVA;   Morquio A Syndrome
Intervention:
10 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
11 Recruiting A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Conditions: Mucopolysaccharidosis Type IIIB;   Mucopolysaccharidosis Type 3 B;   MPS III B;   MPS 3 B
Intervention:
12 Completed Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
Conditions: Mucopolysaccharidosis IV A;   Mucopolysaccharidosis VI
Intervention: Other: Enzyme testing
13 Withdrawn Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa
Conditions: Mucopolysaccharidosis IV;   Morquio A Disease
Intervention:
14 Unknown † Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Conditions: Mucopolysaccharidosis IVA;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: BMN 110
15 Recruiting Mucopolysaccharidosis I (MPS I) Registry
Condition: Mucopolysaccharidosis I (MPS I)
Intervention:
16 Completed
Has Results 		Study of rhASB in Patients With Mucopolysaccharidosis VI
Condition: Mucopolysaccharidosis VI
Interventions: Drug: N-acetylgalactosamine 4-sulfatase;   Drug: Placebo/rhASB
17 Completed
Has Results 		A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
Conditions: Mucopolysaccharidosis I;   Hurler's Syndrome;   Hurler-Scheie Syndrome;   Scheie Syndrome
Intervention: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
18 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
19 Terminated Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I
Conditions: Spinal Cord Compression;   Mucopolysaccharidosis I;   Hurler-Scheie Syndrome;   Scheie Syndrome;   Lysosomal Storage Disease
Intervention: Drug: laronidase
20 Completed
Has Results 		A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Condition: MPS IV A
Interventions: Drug: BMN 110 Weekly;   Drug: Placebo;   Drug: BMN 110 Every Other Week
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† Indicates status has not been verified in more than two years