Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
94 studies found for:    "mucopolysaccharidosis type IV"
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Rank Status Study
1 Completed Psychological Concomitants of Morquio Syndrome (The MAP Study)
Conditions: Morquio Disease;   Mucopolysaccharidosis IV
Intervention:
2 Enrolling by invitation Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
Conditions: Morquio Syndrome A;   Maroteaux Lamy Syndrome;   MPS IVA;   MPS VI
Intervention:
3 Recruiting Biomarker for Morquio Disease
Conditions: Lysosomal Storage Diseases;   Morquio Disease
Intervention:
4 Suspended Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa
Conditions: Mucopolysaccharidosis IV;   Morquio A Disease
Intervention:
5 Recruiting Gait Analysis in MPS IVA
Conditions: MPS IVA;   Morquio Syndrome
Intervention:
6 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
7 Terminated Morquio's Syndrome: a Case Study
Condition: Morquio's Disease
Intervention: Other: physiotherapeutic treatment
8 Recruiting A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Conditions: Mucopolysaccharidosis IV Type A;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: Vimizim® (elosulfase alfa)
9 Terminated Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
Condition: Mucopolysaccharidosis Type IVA
Intervention:
10 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
11 Recruiting Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)
Conditions: Morquio A Syndrome;   Mucopolysaccharidosis IV A
Intervention:
12 Terminated Safety and Exercise Study of BMN 110 for Morquio A Syndrome
Conditions: Mucopolysaccharidosis IVA;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: BMN 110
13 Active, not recruiting 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
14 Terminated Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
Conditions: Mucopolysaccharidosis IVA;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: BMN 110
15 Approved for marketing BMN 110 US Expanded Access Program
Conditions: Mucopolysaccharidosis IVA;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: BMN 110
16 Recruiting Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
Conditions: Mucopolysaccharidosis IV A;   Mucopolysaccharidosis VI
Intervention: Other: Enzyme testing
17 Terminated A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Conditions: MPS IV A;   Mucopolysaccharidosis IVA;   Morquio A Syndrome
Intervention:
18 Terminated 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
19 Completed
Has Results
A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
Condition: MPS IV A
Intervention: Drug: BMN 110
20 Available Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant
Conditions: Acute Lymphoblastic Leukemia;   Acute Myeloid Leukemia;   Chronic Myeloid Leukemia;   Myelodysplastic Syndrome;   Lymphomas;   Bone Marrow Failure;   Hemoglobinopathy;   Immune Deficiency;   Osteopetrosis;   Cytopenias;   White Blood Cell Abnormalities;   Red Blood Cell Abnormalities
Intervention: Biological: CD34+ enriched, T Cell Depleted donor stem cell product

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Indicates status has not been verified in more than two years