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Results for “Mucopolysaccharidosis IVA”

23 trials

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Showing 20 of 23 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
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
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 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 applicableEnded earlyNCT01675674
What this trial is testing

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

Who this might be right for
MucopolysaccharidosesMucopolysaccharidosis IMucopolysaccharidosis II+2 more
National MPS Society 3,000
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
Early research (Phase 1)Looking for participantsNCT04532047
What this trial is testing

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Who this might be right for
MPS IMPS IIMPS IVA+6 more
University of California, San Francisco 10
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
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 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
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
Testing effectiveness (Phase 2)Study completedNCT00884949
What this trial is testing

Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA

Who this might be right for
MPS IV A
BioMarin Pharmaceutical 20
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
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