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Osteochondrodysplasias - 25 Studies Found
Estado | Estudiar |
Active, not recruiting |
Nombre del estudio: Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study) Condición:
Fecha: 2014-08-01 |
Completed |
Nombre del estudio: Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease Condición:
Fecha: 2012-10-12 Intervenciones: Other: Enzyme testing Leukocyte activity measurement of Arylsulfatase B and N acetyl galactosamine 6 sul |
Recruiting |
Nombre del estudio: A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) Condición:
Intervenciones: Drug: Vimizim® (elosulfase alfa) Recombinant human N acetylgalactosamine 6 sulfatase (rhGALNS) |
Terminated |
Nombre del estudio: Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics Condición:
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Completed |
Nombre del estudio: Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) Condición:
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Recruiting |
Nombre del estudio: Longitudinal Studies of Brain Structure and Function in MPS Disorders Condición:
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Completed |
Nombre del estudio: A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA Condición: MPS IV A Fecha: 2009-04-10 Intervenciones: Drug: BMN 110 Subjects will receive a weekly 4- to 5-hour |
Completed |
Nombre del estudio: A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) Condición: MPS IV A Fecha: 2011-01-10 Intervenciones:
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Approved for marketing |
Nombre del estudio: BMN 110 US Expanded Access Program Condición:
Fecha: 2013-05-15 Intervenciones: Drug: BMN 110 |
Enrolling by invitation |
Nombre del estudio: Immunodeficiency in Cartilage-hair Hypoplasia: Sub-project on Safety of Vaccination Against Chickenpox Condición: Cartilage-hair Hypoplasia Fecha: 2015-02-10 Intervenciones: Biological: Varilrix Vaccination against varicella in selected cartilage-hair hypoplasia patients |