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Results for “GAA Deficiency”

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Showing 20 of 0 results

Not applicableUnknownNCT01914536
What this trial is testing

A Natural History Study of Adult Onset Pompe Disease Using Muscle MRI

Who this might be right for
Pompe Disease
Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau 30
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
Post-approval studies (Phase 4)Study completedNCT00486889
What this trial is testing

Growth and Development Study of Alglucosidase Alfa

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease
Genzyme, a Sanofi Company 12
Post-approval studies (Phase 4)Study completedNCT01288027
What this trial is testing

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

Who this might be right for
Pompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 16
Not applicableStudy completedNCT00731081
What this trial is testing

Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®

Who this might be right for
Pompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 8
Testing effectiveness (Phase 2)Study completedNCT00053573
What this trial is testing

RhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)

Who this might be right for
Glycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 20
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
Large-scale testing (Phase 3)Study completedNCT00125879
What this trial is testing

Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 16
Testing effectiveness (Phase 2)Active Not RecruitingNCT03019406
What this trial is testing

Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

Who this might be right for
Glycogen Storage Disease Type II-Pompe's Disease
Genzyme, a Sanofi Company 22
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 applicableActive Not RecruitingNCT03694561
What this trial is testing

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)GAA Deficiency
Duke University 20
Testing effectiveness (Phase 2)Study completedNCT00051935
What this trial is testing

The Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

Who this might be right for
Glycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 2
Testing effectiveness (Phase 2)WithdrawnNCT01656590
What this trial is testing

High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II
Columbia University
Testing effectiveness (Phase 2)Study completedNCT00765414
What this trial is testing

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

Who this might be right for
Pompe Disease Late-OnsetGlycogen Storage Disease Type II GSD II
Genzyme, a Sanofi Company 1
Post-approval studies (Phase 4)Study completedNCT00455195
What this trial is testing

Late-Onset Treatment Study Extension Protocol

Who this might be right for
Pompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD-II)Glycogenesis Type II+1 more
Genzyme, a Sanofi Company 81
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
Post-approval studies (Phase 4)Study completedNCT04676373
What this trial is testing

Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen

Who this might be right for
Pompe's Disease
Genzyme, a Sanofi Company 41
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
Not applicableUnknownNCT02708784
What this trial is testing

An MRI Study on Muscular Diseases -Pompe Disease and Dystrophia Myotonica-

Who this might be right for
Glycogen Storage Disease Type 2Dystrophia Myotonica
University of Aarhus 60
Not applicableLooking for participantsNCT00567073
What this trial is testing

Pompe Pregnancy Sub-Registry

Who this might be right for
Glycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-onset)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 20
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