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Results for “Glycogen Storage Disease Type II-Pompe's Disease”

168 trials

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Showing 20 of 168 results

Testing effectiveness (Phase 2)Study completedNCT01380743
What this trial is testing

Drug-drug Interaction Study

Who this might be right for
Pompe Disease
Amicus Therapeutics 25
Not applicableUnknownNCT05272969
What this trial is testing

Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Inclusion Body MyositisSpinal Muscular Atrophy Type 3+1 more
LMU Klinikum 95
Not applicableApproved For MarketingNCT00074919
What this trial is testing

Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type IIGlycogenosis 2
Genzyme, a Sanofi Company
Not applicableUnknownNCT04755751
What this trial is testing

Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease.

Who this might be right for
Pompe Disease Infantile-Onset
Rambam Health Care Campus 4
Not applicableLooking for participantsNCT05989620
What this trial is testing

Long-Term Development of Muscular Dystrophy Outcome Assessments

Who this might be right for
LGMD1BLGMD1CLGMD1D+26 more
Virginia Commonwealth University 1,000
Not applicableStudy completedNCT04907162
What this trial is testing

Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders

Who this might be right for
Pompe Disease (Late-onset)Myotonic Dystrophy Type 1 (DM1)Myotonic Dystrophy Type 2+4 more
LMU Klinikum 82
Not applicableEnrolling By InvitationNCT07072676
What this trial is testing

The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.

Who this might be right for
Inclusion Body MyositisMyotonic Dystrophy 1Myotonic Dystrophy 2+10 more
LMU Klinikum 30
Testing effectiveness (Phase 2)Study completedNCT01230801
What this trial is testing

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
BioMarin Pharmaceutical 22
Not applicableLooking for participantsNCT04639336
What this trial is testing

Cognitive and Neurological Pathologies in Pompe Disease

Who this might be right for
Pompe Disease
Duke University 45
Large-scale testing (Phase 3)Ended earlyNCT01924845
What this trial is testing

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Who this might be right for
Late-onset Pompe Disease
BioMarin Pharmaceutical 24
Not applicableLooking for participantsNCT07834320
What this trial is testing

Pompe Disease RWE Long-term Monitoring Program

Who this might be right for
Pompe Disease
National Taiwan University Hospital 40
Not applicableStudy completedNCT01758354
What this trial is testing

Newborn Screening Assay of Pompe's Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 236,536
Early research (Phase 1)Study completedNCT03533673
What this trial is testing

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
AskBio Inc 7
Testing effectiveness (Phase 2)Looking for participantsNCT06391736
What this trial is testing

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

Who this might be right for
Pompe Disease (Late-onset)
GeneCradle Inc 33
Not applicableUnknownNCT02120235
What this trial is testing

Investigating Lysosomal Storage Diseases in Minority Groups

Who this might be right for
Lysosomal Storage DisordersGaucher DiseaseFabry Disease+2 more
O & O Alpan LLC 20,000
Not applicableWithdrawnNCT01457443
What this trial is testing

Biomarker for Pompe Disease (BioPompe)

Who this might be right for
Cardiac DiseasesMuscular WeaknessHepato-splenomegaly+4 more
CENTOGENE GmbH Rostock
Not applicableWithdrawnNCT02385162
What this trial is testing

Biomarker for Glycogen Storage Diseases (BioGlycogen)

Who this might be right for
Fructose Metabolism, Inborn ErrorsGlycogen Storage DiseaseGlycogen Storage Disease Type I+7 more
CENTOGENE GmbH Rostock
Early research (Phase 1)Study completedNCT01898364
What this trial is testing

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD II)Acid Maltase Deficiency
Genzyme, a Sanofi Company 24
Not applicableEnded earlyNCT00277979
What this trial is testing

3D Echocardiography Managing Infantile Pompe's Disease

Who this might be right for
Congenital Disorders
Children's Healthcare of Atlanta 2
Large-scale testing (Phase 3)Study completedNCT04138277
What this trial is testing

Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 119
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