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Results for “Glycogen Storage Diseases”

221 trials

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Showing 20 of 221 results

Early research (Phase 1)Study completedNCT03041363
What this trial is testing

Treatment Development of Triheptanoin (G1D)

Who this might be right for
EpilepsyGLUT1DS1Glut1 Deficiency Syndrome 1, Autosomal Recessive+4 more
Juan Pascual 12
Not applicableStudy completedNCT05083806
What this trial is testing

MSOT in Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Pompe's Disease Juvenile Onset+1 more
University of Erlangen-Nürnberg Medical School 20
Not applicableLooking for participantsNCT06813443
What this trial is testing

Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation

Who this might be right for
CardiomyopathiesAmyloidosis CardiacFabry Disease+14 more
IRCCS Azienda Ospedaliero-Universitaria di Bologna 700
Not applicableUnknownNCT05272969
What this trial is testing

Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Inclusion Body MyositisSpinal Muscular Atrophy Type 3+1 more
LMU Klinikum 95
Testing effectiveness (Phase 2)Ended earlyNCT04990388
What this trial is testing

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

Who this might be right for
Glycogen Storage Disease Type III
Ultragenyx Pharmaceutical Inc 9
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
Testing effectiveness (Phase 2)Study completedNCT02919631
What this trial is testing

Triheptanoin in Mc Ardle

Who this might be right for
Glycogen Storage Disease Type V
Institut National de la Santé Et de la Recherche Médicale, France 12
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
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 applicableUnknownNCT00713453
What this trial is testing

Muscle Response to Enzyme Replacement Therapy in Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 16
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 applicableLooking for participantsNCT04639336
What this trial is testing

Cognitive and Neurological Pathologies in Pompe Disease

Who this might be right for
Pompe Disease
Duke University 45
Not applicableEnrolling By InvitationNCT06605612
What this trial is testing

Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders

Who this might be right for
Inclusion Body MyositisMyotonic DystrophyLimb-girdle and Facioscapulohumeral Muscular Dystrophies+9 more
LMU Klinikum 108
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
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
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 completedNCT02318966
What this trial is testing

Glycosade v UCCS in the Dietary Management of Hepatic GSD

Who this might be right for
Glycogen Storage Disease
Vitaflo International, Ltd 61
Not applicableStudy completedNCT04044508
What this trial is testing

Modified Ketogenic Diet in Patients With McArdle Disease Part B

Who this might be right for
McArdle Disease
Rigshospitalet, Denmark 20
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 applicableStudy completedNCT01854242
What this trial is testing

Study of the Relationship Between Glycogen Storage Disease Type Ia and Inflammatory Bowel Disease

Who this might be right for
Inflammatory Bowel DiseaseGlycogen Storage Disease Type Ia
University of Florida 50
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