105 studies found for:    "mucopolysaccharidosis type IV"
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Rank Status Study
1 Recruiting Longitudinal Studies of Brain Structure and Function in MPS Disorders
Conditions: Mucopolysaccharidosis Type I;   Mucopolysaccharidosis Type II;   Mucopolysaccharidosis Type VI;   Mucopolysaccharidosis Type IV;   Mucopolysaccharidosis Type VII
2 Completed Psychological Concomitants of Morquio Syndrome (The MAP Study)
Conditions: Morquio Disease;   Mucopolysaccharidosis IV
3 Terminated Morquio's Syndrome: a Case Study
Condition: Morquio's Disease
Intervention: Other: physiotherapeutic treatment
4 Recruiting Biomarker for Morquio Disease
Conditions: Lysosomal Storage Diseases;   Morquio Disease
5 Completed 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
6 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
7 Recruiting Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)
Conditions: Morquio A Syndrome;   Mucopolysaccharidosis IV A
8 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
9 Terminated A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Conditions: MPS IV A;   Mucopolysaccharidosis IVA;   Morquio A Syndrome
10 Recruiting Gait Analysis in MPS IVA
Conditions: MPS IVA;   Morquio Syndrome
11 Suspended Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa
Conditions: Mucopolysaccharidosis IV;   Morquio A Disease
12 Completed Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
Conditions: Mucopolysaccharidosis IV A;   Mucopolysaccharidosis VI
Intervention: Other: Enzyme testing
13 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)
14 Terminated Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
Condition: Mucopolysaccharidosis Type IVA
15 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
16 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
17 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
18 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
19 Enrolling by invitation Immunodeficiency in Cartilage-hair Hypoplasia: Sub-project on Safety of Vaccination Against Chickenpox
Condition: Cartilage-hair Hypoplasia
Intervention: Biological: Varilrix
20 Completed Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial
Condition: Rubinstein-Taybi Syndrome
Interventions: Drug: sodium valproate;   Drug: Placebo

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Indicates status has not been verified in more than two years