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Mucopolysaccharidosis Type IV - 47 Studies Found
Estado | Estudiar |
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) |
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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Enrolling by invitation |
Nombre del estudio: Identification and Characterization of Bone-related Genetic Variants in Families Condición: Skeletal Dysplasia Fecha: 2015-12-16 Intervenciones: Other: Samples With DNA After consenting, all subjects hav |
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 |
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: 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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Terminated |
Nombre del estudio: Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA Condición: Mucopolysaccharidosis Type IVA Fecha: 2012-11-21 |
Approved for marketing |
Nombre del estudio: BMN 110 US Expanded Access Program Condición:
Fecha: 2013-05-15 Intervenciones: Drug: BMN 110 |
Terminated |
Nombre del estudio: A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) Condición:
Fecha: 2010-10-28 Intervenciones: Drug: BMN 110 Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of |