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Results for “Morquio A Disease”

32 trials

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Showing 20 of 32 results

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
Not applicableApproved For MarketingNCT01858103
What this trial is testing

BMN 110 US Expanded Access Program

Who this might be right for
Mucopolysaccharidosis IVAMorquio A SyndromeMPS IVA
BioMarin Pharmaceutical
Testing effectiveness (Phase 2)Ended earlyNCT01697319
What this trial is testing

Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

Who this might be right for
Mucopolysaccharidosis IVAMorquio A SyndromeMPS IVA
BioMarin Pharmaceutical 16
Not applicableLooking for participantsNCT05619900
What this trial is testing

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more
University of California, San Francisco 250
Not applicableStudy completedNCT01961518
What this trial is testing

Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome

Who this might be right for
Morquio Syndrome AMaroteaux Lamy SyndromeMPS IVA+1 more
Greenwood Genetic Center 17
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 applicableUnknownNCT04624789
What this trial is testing

Registry Gangliosidoses

Who this might be right for
GangliosidosesGM1 GangliosidosisMorquio B Disease+6 more
SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) 40
Not applicableWithdrawnNCT01457456
What this trial is testing

Biomarker for Morquio Disease (BioMorquio)

Who this might be right for
Morquio SyndromeAccumulation of MucopolysaccharidesMorquio Syndrome A+1 more
CENTOGENE GmbH Rostock
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
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
Not applicableEnded earlyNCT00787995
What this trial is testing

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

Who this might be right for
MPS IV AMucopolysaccharidosis IVAMorquio A Syndrome
BioMarin Pharmaceutical 353
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
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
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
Not applicableStudy completedNCT01920828
What this trial is testing

Gait Analysis in MPS IVA

Who this might be right for
MPS IVAMorquio Syndrome
Oregon Health and Science University 10
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
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 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 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 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
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