100 studies found for:    "mucopolysaccharidosis type IV"
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Rank Status Study
21 Approved for marketing BMN 110 US Expanded Access Program
Conditions: Mucopolysaccharidosis IVA;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: BMN 110
22 Terminated 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 Safety and Exercise Study of BMN 110 for Morquio A Syndrome
Conditions: Mucopolysaccharidosis IVA;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: BMN 110
24 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
25 Not yet recruiting BBD Longitudinal Study of Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Intervention:
26 Not yet recruiting Multicenter,Single-arm Study to Evaluate Efficacy, Safety, & Pharmacokinetics of Denosumab in Children w/ OI
Condition: Osteogenesis Imperfecta
Intervention: Drug: Denosumab
27 Completed Efficacy and Safety of Alendronate in Chinese Children or Adolescents With Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Intervention: Drug: Alendronate
28 Recruiting Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Intervention: Biological: Mesenchymal Stem Cells
29 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
30 Active, not recruiting A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
Condition: Achondroplasia
Intervention: Drug: BMN 111
31 Recruiting Natural History of Cherubism Observational Study
Condition: Cherubism
Intervention: Other: no interventions
32 Completed Translational Therapy in Patients With Osteogenesis Imperfecta - A Pilot Trial on Treatment With the Rankl-Antibody Denosumab
Condition: Osteogenesis Imperfecta
Intervention: Drug: Denosumab
33 Unknown  TOCILIZUMAB IN FIBROUS DYSPLASIA OF BONE
Condition: Fibrous Dysplasia of Bone
Interventions: Drug: Tocilizumab;   Drug: Placebo
34 Completed Effect of High-Dose Vitamin D on Bone Density in Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Interventions: Dietary Supplement: standard-dose vitamin D (400IU per day);   Dietary Supplement: high-dose vitamin D (2000 IU per day)
35 Completed Immune Response in the SAPHO Syndrome
Condition: SAPHO Syndrome
Intervention:
36 Recruiting The Effect of Treatment With Teriparatide and Zoledronic Acid in Patients With Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Interventions: Drug: Zoledronic acid;   Drug: Teriparatide;   Drug: placebo zoledronic acid;   Drug: placebo teriparatide
37 Recruiting Genetic and Functional Analysis of Cherubism
Condition: Cherubism
Intervention:
38 Not yet recruiting Growth Hormone for Osteoporosis Pseudoglioma Syndrome
Condition: Osteoporosis Pseudoglioma Syndrome
Intervention: Biological: Human recombinant growth hormone
39 Enrolling by invitation A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
Condition: Achondroplasia
Intervention:
40 Completed A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
Condition: Achondroplasia
Interventions: Drug: BMN 111;   Drug: Normal Saline

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Indicates status has not been verified in more than two years