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Results for “Morquio A Disease”

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Showing 12 of 0 results

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 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
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 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 applicableActive Not RecruitingNCT03655223
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
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 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 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
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 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