Osteochondrodysplasias - 25 Studies Found
Active, not recruiting |
: Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study) :
: 2014-08-01 |
Completed |
: Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease :
: 2012-10-12 : Other: Enzyme testing Leukocyte activity measurement of Arylsulfatase B and N acetyl galactosamine 6 sul |
Recruiting |
: A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) :
: Drug: Vimizim® (elosulfase alfa) Recombinant human N acetylgalactosamine 6 sulfatase (rhGALNS) |
Terminated |
: Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics :
|
Completed |
: Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) :
|
Recruiting |
: Longitudinal Studies of Brain Structure and Function in MPS Disorders :
|
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 |
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 |
Enrolling by invitation |
: Immunodeficiency in Cartilage-hair Hypoplasia: Sub-project on Safety of Vaccination Against Chickenpox : Cartilage-hair Hypoplasia : 2015-02-10 : Biological: Varilrix Vaccination against varicella in selected cartilage-hair hypoplasia patients |