Mucopolysaccharidosis Type IV - 47 Studies Found
Recruiting |
: A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) :
: Drug: Vimizim® (elosulfase alfa) Recombinant human N acetylgalactosamine 6 sulfatase (rhGALNS) |
Completed |
: Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) :
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Recruiting |
: Longitudinal Studies of Brain Structure and Function in MPS Disorders :
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Enrolling by invitation |
: Identification and Characterization of Bone-related Genetic Variants in Families : Skeletal Dysplasia : 2015-12-16 : Other: Samples With DNA After consenting, all subjects hav |
Completed |
: A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA : MPS IV A : 2009-04-10 : Drug: BMN 110 Subjects will receive a weekly 4- to 5-hour |
Terminated |
: Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics :
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Completed |
: A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) : MPS IV A : 2011-01-10 :
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Terminated |
: Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA : Mucopolysaccharidosis Type IVA : 2012-11-21 |
Approved for marketing |
: BMN 110 US Expanded Access Program :
: 2013-05-15 : Drug: BMN 110 |
Terminated |
: A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) :
: 2010-10-28 : Drug: BMN 110 Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of |