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Results for “Glycogen Storage Disease Type II (GSD-II)”

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Showing 20 of 0 results

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
Not applicableUnknownNCT05448131
What this trial is testing

ERT in Pompe Disease: Elucidation of Molecular Structures Contributing to Enzyme Uptake and Immunoreactivity

Who this might be right for
Pompe-Disease
Centre for Analytical Biochemistry and Biomedical Mass Spectrometry 50
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 applicableStudy completedNCT02903654
What this trial is testing

Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French Guiana

Who this might be right for
Pompe's Disease
Centre Hospitalier de Cayenne 925
Not applicableLooking for participantsNCT05619900
What this trial is testing

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more
University of California, San Francisco 250
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 completedNCT00074932
What this trial is testing

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

Who this might be right for
Glycogen Storage Disease Type IIGlycogenosis 2
Genzyme, a Sanofi Company 9
Not applicableLooking for participantsNCT06121011
What this trial is testing

A Global Prospective Observational Registry of Patients With Pompe Disease

Who this might be right for
Pompe Disease
Amicus Therapeutics 500
Testing effectiveness (Phase 2)Active Not RecruitingNCT04093349
What this trial is testing

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+4 more
Spark Therapeutics, Inc. 4
Not applicableStudy completedNCT05431127
What this trial is testing

High Dose Inspiratory Muscle Training in LOPD

Who this might be right for
Late-Onset Pompe DiseaseLysosomal Disease
Duke University 34
Not applicableStudy completedNCT01451879
What this trial is testing

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

Who this might be right for
Pompe Disease
University of Florida 11
Not applicableStudy completedNCT00077662
What this trial is testing

A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 61
Not applicableEnded earlyNCT02221362
What this trial is testing

A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

Who this might be right for
Late-onset Pompe Patients Untreated or Treated With rhGAA
BioMarin Pharmaceutical 30
Not applicableActive Not RecruitingNCT05943678
What this trial is testing

Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study.

Who this might be right for
Pompe DiseaseMcArdle Disease
Medical University of Vienna 30
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
Early research (Phase 1)Looking for participantsNCT07354724
What this trial is testing

Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

Who this might be right for
Late-onset Pompe Disease
Denali Therapeutics Inc. 32
Not applicableStudy completedNCT02354651
What this trial is testing

Response to Diaphragmatic Pacing in Subjects With Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 9
Not applicableUnknownNCT05951790
What this trial is testing

Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease

Who this might be right for
Glycogen Storage Disease Type IIPompe Disease (Late-onset)
Fondazione Don Carlo Gnocchi ETS 20
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 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
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