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Results for “Pompe Disease”

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Showing 20 of 0 results

Not applicableStudy completedNCT03893240
What this trial is testing

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+3 more
Spark Therapeutics, Inc. 61
Large-scale testing (Phase 3)Active Not RecruitingNCT03911505
What this trial is testing

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 21
Not applicableUnknownNCT02761421
What this trial is testing

Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT

Who this might be right for
Glycogen Storage Disease Type II
Taipei Veterans General Hospital, Taiwan 15
Testing effectiveness (Phase 2)Ended earlyNCT01435772
What this trial is testing

Extension Study for Patients Who Have Participated in a BMN 701 Study

Who this might be right for
Pompe Disease
BioMarin Pharmaceutical 21
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 completedNCT07685314
What this trial is testing

LATE-ONSET POMPE DISEASE AND CEREBROVASCULAR MANIFESTATIONS

Who this might be right for
Late-Onset Pompe Disease
Hospitales Universitarios Virgen del Rocío 477
Not applicableStudy completedNCT03147664
What this trial is testing

Cardiopulmonary Exercise Test to Quantify Enzyme Replacement Response in Pediatric Pompe Disease

Who this might be right for
Enzyme Replacement Therapy in Pompe Disease
Rambam Health Care Campus 5
Large-scale testing (Phase 3)Study completedNCT00268944
What this trial is testing

Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support

Who this might be right for
Pompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 5
Not applicableStudy completedNCT02950298
What this trial is testing

Pompe Telemedicine Developmental Study

Who this might be right for
Pompe DiseaseGlycogen Storage Disease II
Duke University 22
Not applicableStudy completedNCT02654886
What this trial is testing

Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.

Who this might be right for
Pompe DiseaseMuscle Weakness
University of California, Irvine 10
Not applicableUnknownNCT02635269
What this trial is testing

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Who this might be right for
Metabolism, Inborn ErrorsLipid Metabolism, Inborn ErrorsCarbohydrate Metabolism, Inborn Errors+20 more
Rigshospitalet, Denmark 60
Not applicableLooking for participantsNCT02399748
What this trial is testing

A Long-term Study for the Outcome of Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 50
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 applicableApproved For MarketingNCT00520143
What this trial is testing

Alglucosidase Alfa Temporary Access Program

Who this might be right for
Glycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-Onset)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company
Testing effectiveness (Phase 2)Study completedNCT00250939
What this trial is testing

RhGAA in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 5
Testing effectiveness (Phase 2)UnknownNCT06130228
What this trial is testing

Nutritional Therapy in Late-onset Pompe Disease

Who this might be right for
Pompe DiseaseMuscle LossObesity+5 more
McMaster University 28
Not applicableUnknownNCT04476550
What this trial is testing

Clinical Specimen Collection From Pompe Disease Patients

Who this might be right for
Pompe Disease
Serhat Gumrukcu, MD PhD 12
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 applicableLooking for participantsNCT00231400
What this trial is testing

Pompe Disease Registry Protocol

Who this might be right for
Glycogen Storage Disease Type IIPompe Disease
Genzyme, a Sanofi Company 2,000
Not applicableUnknownNCT01482494
What this trial is testing

Detection of Pompe Disease in Adult Patients With Myopathies of Uncertain Origin or With Asymptomatic Hyper-CK-emia

Who this might be right for
Pompe Disease
Jordi Perez Lopez 50
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