95 studies found for:    "Osteochondrodysplasias"
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Rank Status Study
1 Completed 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 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:
5 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
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 Recruiting A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Condition: MPS IV A
Intervention:
8 Recruiting Gait Analysis in MPS IVA
Conditions: MPS IVA;   Morquio Syndrome
Intervention:
9 Not yet recruiting Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa
Conditions: Mucopolysaccharidosis IV;   Morquio A Disease
Intervention:
10 Recruiting Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
Conditions: Mucopolysaccharidosis IV A;   Mucopolysaccharidosis VI
Intervention: Other: Enzyme testing
11 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
12 Recruiting Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
Condition: Mucopolysaccharidosis Type IVA
Intervention:
13 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
14 Active, not recruiting 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
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 Active, not recruiting Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial
Condition: Rubinstein-Taybi Syndrome
Interventions: Drug: sodium valproate;   Drug: Placebo
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 MT2013-31:Allo BMT for Metabolic Disorders, Osteopetrosis and Males With Rett Syndrome
Conditions: Mucopolysaccharidosis I;   Mucopolysaccharidosis II;   Mucopolysaccharidosis VI;   Mucopolysaccharidosis VII;   Hurler Syndrome;   Hunter Syndrome;   Maroteaux Lamy Syndrome;   Sly Syndrome;   Glycoprotein Metabolic Disorders;   Alpha Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Adrenoleukodystrophy;   Peroxisomal Disorders;   Osteopetrosis;   Rett Syndrome;   Sphingolipidosis;   Gangliosidosis;   Globoid Cell Leukodystrophy;   Metachromatic Leukodystrophy;   Niemann Pick B;   Niemann Pick C Subtype 2;   I-cell Disease
Interventions: Procedure: blood stem cell transplant;   Drug: Rabbit Anti-Thymocyte Globulin (ATG);   Drug: Fludarabine;   Drug: Busulfan;   Drug: Cyclophosphamide;   Drug: Cyclosporine A (CSA);   Drug: Methylprednisolone;   Drug: Mycophenolate Mofetil (MMF);   Drug: Granulocyte-Colony Stimulating Factor (G-CSF);   Drug: Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF);   Drug: N-acetylcysteine;   Drug: Celecoxib;   Drug: Vitamin E;   Drug: Alpha Lipoic Acid

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Indicates status has not been verified in more than two years