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Clinical Trials on Pompe Disease Infantile-Onset
Total 900 results
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University of Erlangen-Nürnberg Medical SchoolRecruitingPompe Disease (Late-onset) | Pompe Disease | Pompe's Disease Juvenile Onset | Pompe Disease Infantile-OnsetGermany
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GeneCradle IncRecruitingPompe Disease Infantile-OnsetChina
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Seventh Medical Center of PLA General HospitalGeneCradle Therapeutics, IncRecruiting
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Rambam Health Care CampusRecruitingPompe Disease Infantile-OnsetIsrael
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Amicus TherapeuticsAvailablePompe Disease Infantile-OnsetTaiwan, United States, Italy
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Genzyme, a Sanofi CompanyTerminatedGlycogenosis 2 | Glycogen Storage Disease Type II (GSD II) | Acid Maltase Deficiency | Pompe Disease (Infantile-Onset)United States, Taiwan, Germany
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Assiut UniversityUnknownStudy Genetic Basis of Infantile Onset Diabetes Mellitus
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Pompe Disease Infantile-OnsetUnited States, France, South Africa
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Shimaa KamalActive, not recruitingGene Mutation in Infantile Onset Diabetes MellitusEgypt
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University of California, San FranciscoDuke UniversityRecruitingWolman Disease | MPS IVA | Pompe Disease Infantile-Onset | Gaucher Disease, Type 2 | MPS VI | MPS I | Gaucher Disease, Type 3 | MPS II | Mps VIIUnited States
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GeneCradle IncRecruitingPompe Disease (Late-onset)China
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Fundació Institut de Recerca de l'Hospital de la...Genzyme, a Sanofi CompanyUnknownPompe Disease | Late Onset Pompe DiseaseSpain
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Astellas Gene TherapiesRecruitingPompe Disease (Late-onset)United States, Spain, France, Taiwan, Italy
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Amicus TherapeuticsCompletedPompe Disease (Late-onset)United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
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Duke UniversityCompleted
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Amicus TherapeuticsTerminatedLate-onset Pompe DiseaseUnited States, Canada, Australia, Belgium
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BioMarin PharmaceuticalTerminatedLate-onset Pompe DiseaseUnited States, United Kingdom, Germany, France, Italy, Portugal, Belgium, Netherlands
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Huashan HospitalNot yet recruitingPompe Disease (Late-onset)China
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Astellas Gene TherapiesRecruitingPompe Disease (Late-onset)United States, United Kingdom
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Amicus TherapeuticsRecruitingPompe Disease (Late-onset)United States, Taiwan, Japan, Australia, Canada
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Amicus TherapeuticsActive, not recruitingPompe Disease (Late-onset)United States, Germany, Taiwan, Belgium, Australia, Slovenia, Spain, Netherlands, Hungary, Japan, Austria, United Kingdom, France, Argentina, Canada, Sweden, Denmark, New Zealand, Greece, Korea, Republic of, Bosnia and Herzegovina, Ita... and more
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Duke UniversityGenzyme, a Sanofi CompanyActive, not recruitingPompe Disease (Late-onset) | Pompe Disease | GAA DeficiencyUnited States
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BioMarin PharmaceuticalTerminatedLate-onset Pompe DiseaseUnited States, United Kingdom
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Duke UniversityWithdrawnPompe Disease (Late-onset)United States
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BioMarin PharmaceuticalTerminatedLate-onset Pompe Patients Untreated or Treated With rhGAASpain, Slovenia, Serbia, Korea, Republic of, Greece, Australia, Singapore, Brazil, Canada, Taiwan, Ireland, Poland, Romania
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Duke UniversityGenzyme, a Sanofi CompanyCompletedNeuropathy | Myopathy | Glycogen Storage Disease Type II (Late-onset Pompe Disease)United States
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Rigshospitalet, DenmarkDanish Research Center for Magnetic ResonanceRecruitingGlycogen Storage Disease | Pompe Disease (Late-onset) | McArdle DiseaseDenmark
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Spark TherapeuticsActive, not recruitingLysosomal Storage Diseases | Glycogen Storage Disease Type II | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase DeficiencyUnited States, Canada, Netherlands, France, Denmark, Germany, Italy, United Kingdom
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Spark TherapeuticsCompletedLysosomal Storage Diseases | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase DeficiencyUnited States, Italy, United Kingdom, Netherlands, France, Germany
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Assistance Publique - Hôpitaux de ParisNot yet recruitingInterstitial Lung and Liver Disease | Infantile Liver Failure Syndrome 1 | Neurologic, Endocrine and Pancreatic Disease, Multisystem, Infantile-Onset 2 | Rajab Interstitial Lung Disease With Brain Calcifications 2France
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Duke UniversityGenzyme, a Sanofi CompanyRecruitingLate-Onset Pompe Disease | Lysosomal DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease (Late-Onset) | Glycogenesis Type II | Acid Maltase Deficiency (AMD)United States, France, Canada, Netherlands, Australia
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Genzyme, a Sanofi CompanyCompletedGlycogenesis 2 Acid Maltase Deficiency | Pompe Disease (Late-Onset) | Glycogen Storage Disease Type II (GSD II)United States, Netherlands, United Kingdom, Germany
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Genzyme, a Sanofi CompanyCompletedGlycogenesis 2 Acid Maltase Deficiency | Pompe Disease (Late-Onset) | Glycogen Storage Disease Type II (GSD II)France
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Growing Spine Study GroupGrowing Spine FoundationCompletedEarly Onset Scoliosis | Thoracic Insufficiency Syndrome | Congenital Spinal Deformities | Infantile Idiopathic Scoliosis | Neuromuscular Spinal Deformity | Syndromic Spinal DeformityUnited States, Canada, United Kingdom, Egypt, Ghana, Spain, Turkey
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Genzyme, a Sanofi CompanyRecruitingPompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Glycogenesis 2 Acid Maltase DeficiencyCroatia, United States, Italy, Belgium, Czechia
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Assistance Publique - Hôpitaux de ParisOSO-AIRecruiting
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SOFAR S.p.A.CompletedInfantile Colic | Colic, InfantileItaly
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Yuzuncu Yıl UniversityIstanbul University - Cerrahpasa (IUC)Completed
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Federico II UniversityCompleted
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Fondazione Don Carlo Gnocchi OnlusAssociazione Italiana Glicogenosi (AIG); Associazione Riabilitatori Insufficienza... and other collaboratorsRecruitingGlycogen Storage Disease Type II | Pompe Disease (Late-onset)Italy
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Clinic for Special ChildrenRecruitingMyopathy | Myopathy; Hereditary | Myopathies, Nemaline | TNNT1-associated Myopathy | Infantile-onset Nemaline Rod Myopathy | Myopathy, Rod | Amish Nemaline Myopathy | Nemaline Myopathy 5 | NEM5 | Genetic Muscle Disease | Recessive Hereditary Disorder (Autosomal) | ANMUnited States
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Genzyme, a Sanofi CompanyCompletedPompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2Netherlands
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Genzyme, a Sanofi CompanyCompletedPompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2France
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Children's Hospital of Orange CountyRecruiting
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CerecinActive, not recruiting
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University of Colorado, DenverPediatric Epilepsy Research Foundation; West Therapuetics, IncSuspended
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Suvasini SharmaUnknownInfantile SpasmIndia
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Genzyme, a Sanofi CompanyCompletedPompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2United States, France, Netherlands
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BiogenNo longer availableInfantile-onset Spinal Muscular AtrophyNew Zealand, Colombia, Turkey