Status | Study |
Recruiting |
Study Name: Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them? Condition: Morquio Disease MPS - Mucopolysaccharidosis Date: 2017-05-10 Interventions: Other: Interview In-person or telephone in-depth interview |
Enrolling by invitation |
Study Name: Identification and Characterization of Bone-related Genetic Variants in Families Condition: Skeletal Dysplasia Date: 2015-12-16 Interventions: Other: Samples With DNA After consenting, all subjects hav |
Enrolling by invitation |
Study Name: Immunodeficiency in Cartilage-hair Hypoplasia: Sub-project on Safety of Vaccination Against Chickenpox Condition: Cartilage-hair Hypoplasia Date: 2015-02-10 Interventions: Biological: Varilrix Vaccination against varicella in selected cartilage-hair hypoplasia patients |
Recruiting |
Study Name: A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) Condition: Mucopolysaccharidosis IV Type A Morquio A Syndrome Date: 2014-10-28 Interventions: Drug: Vimizim® (elosulfase alfa) Recombinant human N acetylgalactosamine 6 sulfatase (rhGALNS) |
Active, not recruiting |
Study Name: Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study) Condition: Morquio A Syndrome Mucopolysaccharidosis IV A Date: 2014-08-01 |
Withdrawn |
Study Name: Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa Condition: Mucopolysaccharidosis IV Morquio A Disease Date: 2014-05-28 |
Completed |
Study Name: Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome Condition: Morquio Syndrome A Maroteaux Lamy Syndrome Date: 2013-10-08 |
Enrolling by invitation |
Study Name: BMN 110 Phase 3B in Australian Patients Condition: Mucopolysaccharidosis IVA (Morquio A Syndrome) Date: 2013-09-10 Interventions: Drug: BMN 110 All pateints receive treatment with BMN 110 |
Completed |
Study Name: Gait Analysis in MPS IVA Condition: MPS IVA Morquio Syndrome Date: 2013-08-08 |
Approved for marketing |
Study Name: BMN 110 US Expanded Access Program Condition: Mucopolysaccharidosis IVA Morquio A Syndrome MPS IVA Date: 2013-05-15 Interventions: Drug: BMN 110 |