108 studies found for:    "mucopolysaccharidosis type IV"
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Rank Status Study
21 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
22 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
23 Enrolling by invitation Immunodeficiency in Cartilage-hair Hypoplasia: Sub-project on Safety of Vaccination Against Chickenpox
Condition: Cartilage-hair Hypoplasia
Intervention: Biological: Varilrix
24 Completed Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial
Condition: Rubinstein-Taybi Syndrome
Interventions: Drug: sodium valproate;   Drug: Placebo
25 Active, not recruiting BMN 110 Phase 3B in Australian Patients
Condition: Mucopolysaccharidosis IVA (Morquio A Syndrome)
Intervention: Drug: BMN 110
26 Not yet recruiting ACTIMMUNE in Intermediate Osteopetrosis
Condition: Osteopetrosis
Intervention: Drug: Interferon gamma-1b
27 Not yet recruiting Achondroplasia Natural History Multicenter Clinical Study
Condition: Achondroplasia
Intervention:
28 Not yet recruiting Use of ACTIMMUNE in Patients With ADO2
Condition: Autosomal Dominant Osteopetrosis Type 2
Intervention: Drug: ACTIMMUNE
29 Active, not recruiting Efficacy of Bisphosphonates in Patients With Synovitis, Acne, Pustulosis, Hyperostosis, and Osteitis (SAPHO) Syndrome
Condition: SAPHO Syndrome
Intervention: Drug: pamidronate disodium
30 Terminated Effects of a Physical Rehabilitation Program Using the Nintendo Wii on Children With Osteogenesis Imperfecta (OI)
Condition: Osteogenesis Imperfecta
Intervention: Other: Training with Nintendo Wii console
31 Recruiting Urinary Biomarkers of OI Pathobiology
Condition: Osteogenesis Imperfecta
Intervention:
32 Recruiting Natural History, Pathogenesis and Outcome of Melorheostosis A Rare Osteosclerotic Disease
Condition: Rheumatic Disease
Intervention:
33 Recruiting BBD Longitudinal Study of Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Intervention:
34 Recruiting Multicenter,Single-arm Study to Evaluate Efficacy, Safety, & Pharmacokinetics of Denosumab in Children w/ OI
Condition: Osteogenesis Imperfecta
Intervention: Drug: Denosumab
35 Completed Efficacy and Safety of Alendronate in Chinese Children or Adolescents With Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Intervention: Drug: Alendronate
36 Recruiting Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Intervention: Biological: Mesenchymal Stem Cells
37 Recruiting MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
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;   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
38 Enrolling by invitation A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
Condition: Achondroplasia
Intervention: Drug: BMN 111
39 Recruiting Natural History of Cherubism Observational Study
Condition: Cherubism
Intervention: Other: no interventions
40 Completed Translational Therapy in Patients With Osteogenesis Imperfecta - A Pilot Trial on Treatment With the Rankl-Antibody Denosumab
Condition: Osteogenesis Imperfecta
Intervention: Drug: Denosumab

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Indicates status has not been verified in more than two years