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Results for “MPS IV A”

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Showing 11 of 0 results

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
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 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
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
Post-approval studies (Phase 4)Study completedNCT02455622
What this trial is testing

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Who this might be right for
Hunter Syndrome
Shire 21
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
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 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)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
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 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