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118 studies found for:    "mucopolysaccharidosis type IV"
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Rank Status Study
21 Unknown  BMN 110 Phase 3B in Australian Patients
Condition: Mucopolysaccharidosis IVA (Morquio A Syndrome)
Intervention: Drug: BMN 110
22 Terminated
Has Results
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
23 Terminated
Has Results
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
Conditions: Mucopolysaccharidosis IVA;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: BMN 110
24 Unknown  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
25 Completed Study of Skeletal Disorders and Short Stature
Conditions: Developmental Bone Disease;   Dwarfism;   Skeletal Dysplasias
Intervention:
26 Recruiting Interest of Serum Periostin Dosage in Patients With Bone Fibrous Dysplasia
Condition: Bone Fibrous Dysplasia
Intervention: Biological: Blood sampling
27 Unknown  Antioxidant Supplementation in Patients With Kashin-Beck Disease
Condition: Kashin-Beck Disease
Intervention: Dietary Supplement: Biological Antioxidant Supplementation
28 Completed Bone Marrow Injection to Replace Diseased Bone in Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
Condition: Polyostotic Fibrous Dysplasia
Intervention:
29 Completed Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial
Condition: Rubinstein-Taybi Syndrome
Interventions: Drug: sodium valproate;   Drug: Placebo
30 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;   Leukocyte Disorders;   Anemia Due to Intrinsic Red Cell Abnormality
Intervention: Biological: CD34+ enriched, T Cell Depleted donor stem cell product
31 Completed Osteogenesis Imperfecta (OI) Quality of Life Survey Pilot Project 2
Conditions: Osteogenesis Imperfecta;   Brittle Bone Disorders
Intervention: Other: Web-based Survey
32 Unknown  Evaluation of Skeletal Maturity for Slipped Capital Femoral Epiphysis
Condition: Slipped Capital Femoral Epiphysis
Intervention: Radiation: Radiographic skeletal age assessment
33 Recruiting PET Imaging of Phosphodiesterase-4 (PDE4) in Brain and Peripheral Organs of McCune-Albright Syndrome
Condition: Nervous System Disease
Interventions: Other: PET;   Other: MRI
34 Recruiting ACTIMMUNE in Intermediate Osteopetrosis
Condition: Osteopetrosis
Intervention: Drug: Interferon gamma-1b
35 Recruiting Use of ACTIMMUNE in Patients With ADO2
Condition: Autosomal Dominant Osteopetrosis Type 2
Intervention: Drug: ACTIMMUNE
36 Unknown  TOCILIZUMAB IN FIBROUS DYSPLASIA OF BONE
Condition: Fibrous Dysplasia of Bone
Interventions: Drug: Tocilizumab;   Drug: Placebo
37 Completed Clinical Assessment of Patients With High Bone Mass Due to Mutation in Lrp5
Condition: Osteopetrosis
Intervention:
38 Unknown  Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis
Condition: Osteopetrosis
Interventions: Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen);   Procedure: Stem Cell Transplantation;   Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)
39 Active, not recruiting Allogeneic Transplantation For Severe Osteopetrosis
Condition: Severe Osteopetrosis
Interventions: Procedure: umbilical cord blood transplantation;   Drug: Campath-1H;   Radiation: Total Lymphoid Irradiation;   Drug: Cyclophosphamide;   Drug: Busulfan;   Drug: Fludarabine monophosphate;   Procedure: marrow graft transplantation
40 Terminated
Has Results
Reduced Intensity AlloTransplant For Osteopetrosis
Condition: Osteopetrosis
Interventions: Procedure: Stem Cell or Umbilical Cord Blood Transplantation;   Drug: Campath, Busulfan, Clofarabine;   Procedure: Total Lymphoid Irradiation

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Study has passed its completion date and status has not been verified in more than two years.