Search clinical trials for: Late Onset Lysosomal Acid Lipase Deficiency (CESD)
Total 42205 results
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Children's Hospital Medical Center, CincinnatiNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedWolman Disease | Cholesterol Ester Storage Disease | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2United States
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Alexion PharmaceuticalsTerminatedLysosomal Acid Lipase DeficiencyUnited States
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | LAL-Deficiency | Cholesterol Ester Storage Disease (CESD)France, United Kingdom, United States, Canada, Czechia
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD) | LAL-DeficiencyFrance, United States, United Kingdom, Czechia
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD)United States, United Kingdom, France, Canada, Czech Republic, Italy, Poland, Switzerland
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase DeficiencyFrance, Poland, United Kingdom, Spain, Mexico, Turkey, Japan, Australia, Russian Federation, United States, Germany, Italy, Czechia, Argentina, Croatia
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Alexion PharmaceuticalsTerminatedLysosomal Acid Lipase DeficiencyUnited Kingdom, Finland, United States, Italy
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Alexion PharmaceuticalsCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited Kingdom, France, United States, Egypt, Ireland
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Centre Hospitalier Universitaire de LiegeSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaboratorsRecruitingCongenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditionsBelgium
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase DeficiencySpain, Germany, Italy, United States, Croatia, Canada, Russian Federation, Denmark, United Kingdom, Belgium, Mexico, Australia, Netherlands, Brazil, Turkey
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Sanford HealthNational Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaboratorsRecruitingMitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditionsUnited States, Australia
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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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Genzyme, a Sanofi CompanyRecruitingGlycogen Storage Disease Type II | Pompe DiseaseHungary, Croatia, Serbia, United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, China, Colombia, Denmark, France, Germany, Greece, Hong Kong, India, Indonesia, Israel, Italy, Japan, Jordan, Korea, Republic of, Kuwait and more
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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Genzyme, a Sanofi CompanyCompleted
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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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Duke UniversityGenzyme, a Sanofi CompanyActive, not recruitingPompe Disease (Late-onset) | Pompe Disease | GAA DeficiencyUnited States
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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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Genzyme, a Sanofi CompanyCompletedPompe Disease | Glycogen Storage Disease Type II (GSD II) | Acid Maltase DeficiencyUnited States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom
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AlexionCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited States, Canada, United Kingdom, Italy, France
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Genzyme, a Sanofi CompanyCompleted
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type IIUnited States, France, Netherlands
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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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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 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 CompanyCompletedGlycogen Storage Disease Type IIUnited States, France, Germany, Israel, Italy, Netherlands, Taiwan
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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 CompanyApproved for marketingGlycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe Disease (Late-Onset)United States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Glycogenosis 2United States
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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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Genzyme, a Sanofi CompanyCompletedPompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2France
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McMaster UniversityNot yet recruitingObesity | Lysosomal Storage Diseases | Muscle Loss | Glycogen Storage Disease Type II | Pompe Disease | Nutrition Poor | Glycogen Storage Disease Type II, Adult | Glycogen Storage Disease Type II Late Onset
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Fondazione SISA (Societa Italiana per lo Studio...Recruiting
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University Of PerugiaRecruitingObesity | Ketosis | Lysosomal Acid Lipase Deficiency | Ketogenic DietingItaly
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AlexionRecruitingWolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-DeficiencyFrance, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Late Onset Tay-Sachs DiseaseUnited States
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Ultragenyx Pharmaceutical IncActive, not recruitingOrnithine Transcarbamylase (OTC) DeficiencyCanada, United States, France, Spain, United Kingdom
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Albert Einstein College of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditionsUnited States
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CENTOGENE GmbH RostockWithdrawnCholesterol Ester Storage Disease | Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Wolman TypeGermany, India, Sri Lanka
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Ultragenyx Pharmaceutical IncCompletedOrnithine Transcarbamylase (OTC) DeficiencyUnited Kingdom, United States, Canada, Spain
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University of FloridaLacerta TherapeuticsCompletedPompe DiseaseUnited States
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Ultragenyx Pharmaceutical IncTerminatedOrnithine Transcarbamylase DeficiencyUnited States
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Mendel TuchmanChildren's Hospital of Philadelphia; University of California, Los Angeles; Icahn... and other collaboratorsCompletedMethylmalonic Acidemia | Carbamoyl-Phosphate Synthase I Deficiency Disease | Propionic Acidemia, Type I and/or Type II | Ornithine Carbamoyltransferase DeficiencyUnited States
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Ankara UniversityAlexion PharmaceuticalsCompleted
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Alexion PharmaceuticalsTerminated
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Hospices Civils de LyonUnknownPatients Waiting for a Liver Transplant.France
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Hospices Civils de LyonUnknownLiver Post-transplant PatientsFrance
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Alexion PharmaceuticalsNo longer availableLysosomal Acid Lipase Deficiency
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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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Columbia UniversityWithdrawnGlycogen Storage Disease Type IIUnited States