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Results for “Glycogenosis 2”

173 trials

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Showing 20 of 173 results

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 applicableActive Not RecruitingNCT04929002
What this trial is testing

Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases

Who this might be right for
Glycogen Storage DiseaseMcArdle DiseasePompe Disease (Late-onset)
Rigshospitalet, Denmark 50
Testing effectiveness (Phase 2)Looking for participantsNCT06092034
What this trial is testing

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

Who this might be right for
Danon Disease
Rocket Pharmaceuticals Inc. 14
Not applicableAvailableNCT03865836
What this trial is testing

Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

Who this might be right for
Pompe Disease
Amicus Therapeutics
Not applicableStudy completedNCT02354664
What this trial is testing

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 14
Not applicableLooking for participantsNCT03564561
What this trial is testing

Natural History of Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II, Adult
Assistance Publique - Hôpitaux de Paris 20
Not applicableNot Yet RecruitingNCT05017402
What this trial is testing

Higher Dose of Alglucosidase Alpha for Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II
Taipei Veterans General Hospital, Taiwan 36
Post-approval studies (Phase 4)UnknownNCT02525172
What this trial is testing

Immune Modulation Therapy for Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 8
Not applicableUnknownNCT04942912
What this trial is testing

Effect of Enzyme Replacement Therapy in Patients With Juvenile-onset Pompe Disease

Who this might be right for
Pompe's Disease Juvenile Onset
Central Hospital, Nancy, France 10
Very early researchEnded earlyNCT02357225
What this trial is testing

A Pilot Study of Pyridostigmine in Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 2
Not applicableStudy completedNCT00113035
What this trial is testing

Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease

Who this might be right for
Pompe Disease
Genzyme, a Sanofi Company 60
Not applicableStudy completedNCT02824068
What this trial is testing

Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study)

Who this might be right for
Pompe Disease
LMU Klinikum 112
Not applicableStudy completedNCT04724213
What this trial is testing

Development of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe Disease

Who this might be right for
Pompe Disease
University Children's Hospital, Zurich 101
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
Testing effectiveness (Phase 2)Study completedNCT01942590
What this trial is testing

Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

Who this might be right for
Pompe Disease
Dwight Koeberl, M.D., Ph.D. 17
Not applicableUnknownNCT02000310
What this trial is testing

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

Who this might be right for
Lysosomal Storage Disorders
O & O Alpan LLC 80
Testing effectiveness (Phase 2)Study completedNCT02032524
What this trial is testing

Avalglucosidase Alfa Extension Study

Who this might be right for
Glycogen Storage Disease Type II Pompe Disease
Genzyme, a Sanofi Company 19
Early research (Phase 1)Active Not RecruitingNCT03533673
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)Ended earlyNCT02898753
What this trial is testing

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
Valerion Therapeutics, LLC 12
Testing effectiveness (Phase 2)Looking for participantsNCT07282847
What this trial is testing

Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)

Who this might be right for
Pompe Disease (Late-onset)Pompe Disease Late-OnsetLOPD
AskBio Inc 12
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