114 studies found for:    "mucopolysaccharidosis type IV"
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Rank Status Study
21 Completed Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial
Condition: Rubinstein-Taybi Syndrome
Interventions: Drug: sodium valproate;   Drug: Placebo
22 Completed Study of Skeletal Disorders and Short Stature
Conditions: Developmental Bone Disease;   Dwarfism;   Skeletal Dysplasias
Intervention:
23 Active, not recruiting BMN 110 Phase 3B in Australian Patients
Condition: Mucopolysaccharidosis IVA (Morquio A Syndrome)
Intervention: Drug: BMN 110
24 Approved for marketing BMN 110 US Expanded Access Program
Conditions: Mucopolysaccharidosis IVA;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: BMN 110
25 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
26 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
27 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
28 Enrolling by invitation Development of a Non-invasive Assessment of Human Bone Quality Using Spatially Offset Raman Spectroscopy
Conditions: Osteoarthritis;   Osteoporosis;   Osteogenesis Imperfecta;   Rickets / Osteomalacia;   Bone Infection
Intervention: Device: spatially offset Raman spectrometer (SORS)
29 Recruiting Osteogenesis Imperfecta (OI) Quality of Life Survey Pilot Project 2
Conditions: Osteogenesis Imperfecta;   Brittle Bone Disorders
Intervention: Other: Web-based Survey
30 Completed PET Imaging of Phosphodiesterase-4 (PDE4) in Brain and Peripheral Organs of McCune-Albright Syndrome
Condition: Nervous System Disease
Interventions: Other: PET;   Other: MRI
31 Enrolling by invitation A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
Condition: Achondroplasia
Intervention: Drug: BMN 111
32 Recruiting ACTIMMUNE in Intermediate Osteopetrosis
Condition: Osteopetrosis
Intervention: Drug: Interferon gamma-1b
33 Enrolling by invitation Achondroplasia Natural History Multicenter Clinical Study
Condition: Achondroplasia
Intervention:
34 Recruiting Use of ACTIMMUNE in Patients With ADO2
Condition: Autosomal Dominant Osteopetrosis Type 2
Intervention: Drug: ACTIMMUNE
35 Active, not recruiting Efficacy of Bisphosphonates in Patients With Synovitis, Acne, Pustulosis, Hyperostosis, and Osteitis (SAPHO) Syndrome
Condition: SAPHO Syndrome
Intervention: Drug: pamidronate disodium
36 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
37 Recruiting Urinary Biomarkers of OI Pathobiology
Condition: Osteogenesis Imperfecta
Intervention:
38 Recruiting Natural History, Pathogenesis and Outcome of Melorheostosis A Rare Osteosclerotic Disease
Condition: Rheumatic Disease
Intervention:
39 Recruiting BBD Longitudinal Study of Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Intervention:
40 Recruiting Multicenter,Single-arm Study to Evaluate Efficacy, Safety, & Pharmacokinetics of Denosumab in Children w/ OI
Condition: Osteogenesis Imperfecta
Intervention: Drug: Denosumab

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Indicates status has not been verified in more than two years