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Results for “Glycogen Storage Disease”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT04154488
What this trial is testing

Mavorixafor in Participants With Congenital Neutropenia and Chronic Idiopathic Neutropenia Disorders

Who this might be right for
Neutropenia
X4 Pharmaceuticals 32
Not applicableUnknownNCT03812042
What this trial is testing

Screening of Lysosomal Storage Disorders Diseases in Minority Groups

Who this might be right for
Lysosomal Storage Diseases
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 100,000
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Not applicableStudy completedNCT03893240
What this trial is testing

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+3 more
Spark Therapeutics, Inc. 61
Large-scale testing (Phase 3)Active Not RecruitingNCT03911505
What this trial is testing

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 21
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
Not applicableLooking for participantsNCT06795152
What this trial is testing

Rare Glycogen Storage Diseases Natural History Study

Who this might be right for
Glycogen Storage DiseaseGSD Type 0AGSD Type 0B+12 more
Duke University 200
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
Not applicableStudy completedNCT07685314
What this trial is testing

LATE-ONSET POMPE DISEASE AND CEREBROVASCULAR MANIFESTATIONS

Who this might be right for
Late-Onset Pompe Disease
Hospitales Universitarios Virgen del Rocío 477
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 applicableStudy completedNCT03147664
What this trial is testing

Cardiopulmonary Exercise Test to Quantify Enzyme Replacement Response in Pediatric Pompe Disease

Who this might be right for
Enzyme Replacement Therapy in Pompe Disease
Rambam Health Care Campus 5
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
Testing effectiveness (Phase 2)UnknownNCT04138251
What this trial is testing

Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b and G6PC3 Deficiency

Who this might be right for
Glycogen Storage Disease Type IGlucose 6 Phosphatase Deficiency
Cliniques universitaires Saint-Luc- Université Catholique de Louvain 5
Not applicableStudy completedNCT02950298
What this trial is testing

Pompe Telemedicine Developmental Study

Who this might be right for
Pompe DiseaseGlycogen Storage Disease II
Duke University 22
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
Not applicableStudy completedNCT03945370
What this trial is testing

Oral Ketone Body Supplementation in Patients With McArdle Disease

Who this might be right for
McArdle Disease
Rigshospitalet, Denmark 8
Not applicableUnknownNCT02635269
What this trial is testing

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Who this might be right for
Metabolism, Inborn ErrorsLipid Metabolism, Inborn ErrorsCarbohydrate Metabolism, Inborn Errors+20 more
Rigshospitalet, Denmark 60
Not applicableLooking for participantsNCT02399748
What this trial is testing

A Long-term Study for the Outcome of Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 50
Not applicableAvailableNCT05930223
What this trial is testing

Intravenous VAL-1221 Lafora Expanded Access Protocol

Who this might be right for
Lafora Disease
Parasail, LLC
Not applicableWithdrawnNCT01457443
What this trial is testing

Biomarker for Pompe Disease (BioPompe)

Who this might be right for
Cardiac DiseasesMuscular WeaknessHepato-splenomegaly+4 more
CENTOGENE GmbH Rostock
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