Not applicableLooking for participantsNCT03333200What this trial is testingLongitudinal Study of Neurodegenerative DisordersWho this might be right forMLDKrabbe DiseaseALD+24 more University of Pittsburgh 1,500
Not applicableStudy completedNCT03150069What this trial is testingPregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?Who this might be right forMorquio DiseaseMPS - Mucopolysaccharidosis Emory University 18
Testing effectiveness (Phase 2)Ended earlyNCT01242111What this trial is testingEvaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)Who this might be right forMPS IV AMucopolysaccharidosis IVAMorquio A Syndrome BioMarin Pharmaceutical 20
Not applicableLooking for participantsNCT06036693What this trial is testingMPS (RaDiCo Cohort) (RaDiCo-MPS)Who this might be right forMucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis III+5 more Institut National de la Santé Et de la Recherche Médicale, France 1,000
Large-scale testing (Phase 3)Study completedNCT01966029What this trial is testingBMN 110 Phase 3B in Australian PatientsWho this might be right forMucopolysaccharidosis IVA (Morquio A Syndrome) BioMarin Pharmaceutical 13
Not applicableStudy completedNCT01870375What this trial is testingLongitudinal Studies of Brain Structure and Function in MPS DisordersWho this might be right forMucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type VI+2 more University of Minnesota 100
Large-scale testing (Phase 3)Study completedNCT01415427What this trial is testingLong-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)Who this might be right forMucopolysaccharidosis IV AMorquio A SyndromeMPS IVA BioMarin Pharmaceutical 173
Not applicableStudy completedNCT02067650What this trial is testingUltrasound Findings of Finger, Wrist and Knee Joints in MucopolysaccharidosisWho this might be right forMPS IMPS IV Children's Hospital of Eastern Ontario 18
Not applicableLooking for participantsNCT05284006What this trial is testingNon-invasive Functional Assessment and Pathogenesis of Morquio AWho this might be right forMucopolysaccharidosis IV Type AMorquio A SyndromeMPS IVA Nemours Children's Clinic 60
Post-approval studies (Phase 4)Study completedNCT00239083What this trial is testingEfficacy and Safety of Enteric-Coated Mycophenolate Sodium (EC-MPS) in Renal Transplant PatientsWho this might be right forKidney Transplantation Novartis 40
Not applicableStudy completedNCT05687474What this trial is testingBaby Detect : Genomic Newborn ScreeningWho this might be right forCongenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more Centre Hospitalier Universitaire de Liege 6,824
Not applicableLooking for participantsNCT07361536What this trial is testingCardiac Structure and Function in MPSWho this might be right forMPS IMPS IV AMPS - Mucopolysaccharidosis+1 more Children's Hospital of Orange County 240
Not applicableEnrolling By InvitationNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Large-scale testing (Phase 3)Study completedNCT01275066What this trial is testingStudy to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)Who this might be right forMPS IV A BioMarin Pharmaceutical 177
Early research (Phase 1)Looking for participantsNCT04532047What this trial is testingPEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)Who this might be right forMPS IMPS IIMPS IVA+6 more University of California, San Francisco 10
Early research (Phase 1)Study completedNCT01173016What this trial is testingAdministration of IV Laronidase Post Bone Marrow Transplant in HurlerWho this might be right forHurler Syndrome Masonic Cancer Center, University of Minnesota 11
Not applicableStudy completedNCT02294877What this trial is testingA Multicenter, Multinational, Observational Morquio A Registry Study (MARS)Who this might be right forMucopolysaccharidosis IV Type AMorquio A SyndromeMPS IVA BioMarin Pharmaceutical 418
Not applicableEnrolling By InvitationNCT05368038What this trial is testingScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramWho this might be right forAcid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more Albert Einstein College of Medicine 100,000
Testing effectiveness (Phase 2)Study completedNCT01515956What this trial is testingStudy of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)Who this might be right forMucopolysaccharidosis IVAMorquio A SyndromeMPS IVA BioMarin Pharmaceutical 15
Testing effectiveness (Phase 2)Ended earlyNCT01609062What this trial is testingSafety and Exercise Study of Two Doses of BMN 110 for Morquio A SyndromeWho this might be right forMucopolysaccharidosis IVAMorquio A SyndromeMPS IVA BioMarin Pharmaceutical 25