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Results for “MPS IV A”

31 trials

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Showing 20 of 31 results

Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
Testing effectiveness (Phase 2)Ended earlyNCT01242111
What this trial is testing

Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Who this might be right for
MPS IV AMucopolysaccharidosis IVAMorquio A Syndrome
BioMarin Pharmaceutical 20
Large-scale testing (Phase 3)Study completedNCT01966029
What this trial is testing

BMN 110 Phase 3B in Australian Patients

Who this might be right for
Mucopolysaccharidosis IVA (Morquio A Syndrome)
BioMarin Pharmaceutical 13
Not applicableStudy completedNCT01870375
What this trial is testing

Longitudinal Studies of Brain Structure and Function in MPS Disorders

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type VI+2 more
University of Minnesota 100
Large-scale testing (Phase 3)Study completedNCT01415427
What this trial is testing

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Who this might be right for
Mucopolysaccharidosis IV AMorquio A SyndromeMPS IVA
BioMarin Pharmaceutical 173
Not applicableStudy completedNCT02067650
What this trial is testing

Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis

Who this might be right for
MPS IMPS IV
Children's Hospital of Eastern Ontario 18
Not applicableLooking for participantsNCT05284006
What this trial is testing

Non-invasive Functional Assessment and Pathogenesis of Morquio A

Who this might be right for
Mucopolysaccharidosis IV Type AMorquio A SyndromeMPS IVA
Nemours Children's Clinic 60
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Not applicableLooking for participantsNCT07361536
What this trial is testing

Cardiac Structure and Function in MPS

Who this might be right for
MPS IMPS IV AMPS - Mucopolysaccharidosis+1 more
Children's Hospital of Orange County 240
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Large-scale testing (Phase 3)Study completedNCT01275066
What this trial is testing

Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Who this might be right for
MPS IV A
BioMarin Pharmaceutical 177
Not applicableStudy completedNCT02294877
What this trial is testing

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

Who this might be right for
Mucopolysaccharidosis IV Type AMorquio A SyndromeMPS IVA
BioMarin Pharmaceutical 418
Not applicableEnrolling By InvitationNCT05368038
What this trial is testing

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Who this might be right for
Acid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more
Albert Einstein College of Medicine 100,000
Testing effectiveness (Phase 2)Study completedNCT01515956
What this trial is testing

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Who this might be right for
Mucopolysaccharidosis IVAMorquio A SyndromeMPS IVA
BioMarin Pharmaceutical 15
Testing effectiveness (Phase 2)Ended earlyNCT01609062
What this trial is testing

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

Who this might be right for
Mucopolysaccharidosis IVAMorquio A SyndromeMPS IVA
BioMarin Pharmaceutical 25
Testing effectiveness (Phase 2)Active Not RecruitingNCT05845749
What this trial is testing

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

Who this might be right for
MPS IVAMPS VI
University of California, San Francisco 6
Not applicableEnded earlyNCT01733615
What this trial is testing

Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA

Who this might be right for
Mucopolysaccharidosis Type IVA
Emory University 3
Not applicableStudy completedNCT03204370
What this trial is testing

Natural History of Atypical Morquio A Disease

Who this might be right for
Mucopolysaccharidosis IV A
GOIZET 7
Not applicableStudy completedNCT02208661
What this trial is testing

Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)

Who this might be right for
Morquio A SyndromeMucopolysaccharidosis IV A
Nadia Ali, PhD 12
Not applicableStudy completedNCT01707433
What this trial is testing

Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease

Who this might be right for
Mucopolysaccharidosis IV AMucopolysaccharidosis VI
Children's Hospitals and Clinics of Minnesota 22
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