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

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Showing 20 of 0 results

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
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
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07750990
What this trial is testing

An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 60
Post-approval studies (Phase 4)Not Yet RecruitingNCT06575829
What this trial is testing

Treatment Frequency Reduction in Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)GAA DeficiencyGlycogen Storage Disease Type II+1 more
Erasmus Medical Center 10
Not applicableStudy completedNCT01701154
What this trial is testing

The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease

Who this might be right for
Pompe Disease
Duke University 11
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
Early research (Phase 1)Ended earlyNCT02185651
What this trial is testing

A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction

Who this might be right for
Pompe DiseaseHypersensitivity Reaction
University of Florida 2
Not applicableLooking for participantsNCT07664930
What this trial is testing

Phrenic Nerve and Diaphragm Electrophysiology in Pompe Disease

Who this might be right for
Pompe Disease
IRCCS National Neurological Institute "C. Mondino" Foundation 20
Not applicableStudy completedNCT02838368
What this trial is testing

Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices

Who this might be right for
Pompe Disease
University of California, Irvine 921
Not applicableStudy completedNCT02746718
What this trial is testing

Frequency of Pompe's Disease and Neuromuscular Etiologies in Patients With Restrictive Respiratory Failure Associated With Signs of Muscle Weakness

Who this might be right for
Pompe Disease
Centre Hospitalier Universitaire de Nice 50
Not applicableLooking for participantsNCT06833489
What this trial is testing

Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases

Who this might be right for
Rare Genetic Muscle DiseasesMuscular Dystrophy, DuchenneMuscular Dystrophy, Becker+2 more
Assistance Publique Hopitaux De Marseille 50
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
Post-approval studies (Phase 4)Ended earlyNCT01597596
What this trial is testing

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Who this might be right for
Pompe Disease (Infantile-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenosis 2+1 more
Genzyme, a Sanofi Company 4
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
Testing effectiveness (Phase 2)Study completedNCT00025896
What this trial is testing

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type IIAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 8
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 applicableStudy completedNCT00830583
What this trial is testing

Pompe Prevalence Study in Patients With Muscle Weakness Without Diagnosis

Who this might be right for
Pompe's Disease
Centre Hospitalier Universitaire de Nice 400
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 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 completedNCT01380743
What this trial is testing

Drug-drug Interaction Study

Who this might be right for
Pompe Disease
Amicus Therapeutics 25
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