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74 studies found for:    "mucopolysaccharidosis type IV"

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Rank Status Study
1 Recruiting Psychological Concomitants of Morquio Syndrome (The MAP Study)
Conditions: Morquio Disease;   Mucopolysaccharidosis IV
Intervention:
2 Recruiting Biomarker for Morquio Disease
Conditions: Lysosomal Storage Diseases;   Morquio Disease
Intervention:
3 Terminated Morquio's Syndrome: a Case Study
Condition: Morquio's Disease
Intervention: Other: physiotherapeutic treatment
4 Recruiting 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
5 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
Intervention: Drug: BMN 110
6 Recruiting A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Condition: MPS IV A
Intervention:
7 Recruiting Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
Conditions: Mucopolysaccharidosis IV A;   Mucopolysaccharidosis VI
Intervention: Other: Enzyme testing
8 Completed 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
9 Recruiting Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
Condition: Mucopolysaccharidosis Type IVA
Intervention:
10 Enrolling by invitation A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Condition: MPS IV A
Intervention: Drug: BMN 110
11 Active, not recruiting 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
12 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
13 Recruiting Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial
Condition: Rubinstein-Taybi Syndrome
Interventions: Drug: sodium valproate;   Drug: Placebo
14 Recruiting 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 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
16 Active, not recruiting Safety and Exercise Study of BMN 110 for Morquio A Syndrome
Conditions: Mucopolysaccharidosis IVA;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: BMN 110
17 Active, not recruiting Pamidronate to Treat Osteogenesis Imperfecta in Children
Condition: Osteogenesis Imperfecta
Intervention: Drug: Pamidronate (Aredia)
18 Completed Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis
Condition: Osteopetrosis
Interventions: Drug: calcitriol;   Drug: interferon gamma
19 Active, not recruiting Arimidex in McCune Albright Syndrome
Condition: McCune-Albright Syndrome
Intervention: Drug: Arimidex 1 mg
20 Recruiting Growth Hormone Therapy in Osteogenesis Imperfecta
Conditions: Dwarfism;   Osteogenesis Imperfecta
Interventions: Drug: Humatrope;   Drug: GRH;   Drug: Nutropin
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† Indicates status has not been verified in more than two years