Mucopolysaccharidosis I - 56 Studies Found
Active, not recruiting |
: Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study) :
: 2014-08-01 |
Withdrawn |
: Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa :
: 2014-05-28 |
Completed |
: Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome :
: 2013-08-08 |
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) :
|
Terminated |
: A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome) :
: 2008-11-06 |
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 :
|
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 |