Clinical Trials on Inherited Metabolic Storage Diseases
Total 61973 results
-
AVROBIOWithdrawn
-
Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited Kingdom, United States
-
Turkish Society of Pediatric Gastroenterology,...CompletedDuchenne Muscular Dystrophy | Pompe Disease (Late-onset)Turkey
-
Baylor Research InstituteUltragenyx Pharmaceutical IncCompletedAdult Polyglucosan Body Disease | Glycogen Brancher Enzyme Deficiency | Glycogen Storage Disease Type IVFrance
-
Assy NimerUnknownNon-alcoholic Fatty Liver Disease | Cholesterol Ester Storage DiseaseIsrael
-
Lysosomal and Rare Disorders Research and Treatment...UnknownGaucher Disease Type 1United States
-
University of PennsylvaniaPassage Bio, Inc.RecruitingGM1 GangliosidosisUnited States, Turkey, Brazil, Canada, United Kingdom
-
National Center for Research Resources (NCRR)University of PittsburghUnknownGaucher's DiseaseUnited States
-
National Institute of Neurological Disorders and...CompletedGaucher's DiseaseUnited States
-
LYSOGENETerminatedGM1 GangliosidosisUnited Kingdom, United States, France
-
LYSOGENECasimir, LLC; Cure GM1 FoundationCompleted
-
Freeline TherapeuticsRecruitingGaucher Disease, Type 1Spain, Israel, United States, United Kingdom, Brazil, Germany, Paraguay
-
The Hospital for Sick ChildrenCompletedGangliosidoses, GM2 | Tay-Sachs Disease | Sandhoff DiseaseCanada
-
National Center for Research Resources (NCRR)National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University...Completed
-
Lysosomal and Rare Disorders Research and Treatment...RecruitingBone Diseases | Gaucher DiseaseUnited States
-
IntraBio IncCompletedGM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Germany, Spain, United Kingdom
-
Terence FlotteMassachusetts General Hospital; University of Massachusetts, WorcesterActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States
-
National Institute of Neurological Disorders and...CompletedGaucher DiseaseUnited States, United Kingdom
-
Centre Hospitalier Universitaire de NiceCompleted
-
Assiut UniversityNot yet recruiting
-
Tehran University of Medical SciencesMashhad University of Medical Sciences; Kashan University of Medical SciencesRecruitingEffects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) (EMTISTD)Supportive Care | GM2 GangliosidosisIran, Islamic Republic of
-
SanofiCompletedGaucher Disease | SplenomegalyFrance
-
The Hospital for Sick ChildrenActelionCompletedGangliosidoses GM2Canada
-
Rambam Health Care CampusUnknown
-
Massachusetts General HospitalActive, not recruiting
-
SanofiCompletedGaucher DiseaseUnited States
-
CENTOGENE GmbH RostockWithdrawnCholesterol Ester Storage Disease | Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Wolman TypeGermany, India, Sri Lanka
-
Massachusetts General HospitalRecruiting
-
CENTOGENE GmbH RostockWithdrawnSkeletal Abnormalities | Psychomotor RetardationGermany, India, Sri Lanka, Egypt
-
Genzyme, a Sanofi CompanyActive, not recruitingGaucher Disease Type 1 | Gaucher Disease Type 3Germany, United States, Japan, United Kingdom
-
KemPharm Denmark A/STerminatedGaucher Disease, Type 1 | Gaucher Disease, Type 3India
-
Baylor Research InstituteTexas Scottish Rite Hospital for ChildrenWithdrawnGaucher Disease Type 1 | Gaucher Disease Type 3United States
-
Assiut UniversityNot yet recruitingPsychiatric Problem | Wolman Disease
-
Rigshospitalet, DenmarkUltragenyx Pharmaceutical IncCompletedTarui Disease | Debrancher Deficiency | GYG1 DEFICIENCYDenmark
-
Hospices Civils de LyonUnknownLiver Post-transplant PatientsFrance
-
Children's Hospital Medical Center, CincinnatiCompletedOsteopenia | Gaucher's DiseaseIsrael, United States
-
SanofiRecruitingGaucher's Disease Type IIIUnited States, Germany, Hungary, France, China, Japan, Canada, Argentina, Italy, Turkey
-
University of British ColumbiaCanadian Institutes of Health Research (CIHR); Saudi Arabian Cultural Bureau...Unknown
-
University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGaucher Disease Type 1United States
-
Children's National Research InstituteActelionCompletedSandhoff Disease | GM2 Gangliosidoses | Tay-SachsUnited States
-
Genzyme, a Sanofi CompanyActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States, Argentina, Czechia, Portugal, Russian Federation, Turkey, United Kingdom, Brazil, France, Germany, Italy, Japan, Spain
-
Azafaros A.G.Active, not recruitingGM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Brazil, France, Germany, Italy, United Kingdom
-
Hospices Civils de LyonUnknownEnd Stage Renal Disease | Fabry Disease | Renal DialysisFrance
-
Genzyme, a Sanofi CompanyCRL/MedinetTerminatedFabry Disease | Chronic Kidney Disease, Stage IV (Severe)United States
-
Cliniques universitaires Saint-Luc- Université...UnknownGlucose 6 Phosphatase Deficiency | Glycogen Storage Disease Type IBelgium
-
Translational Research Center for Medical Innovation...RecruitingNeutral Lipid Storage DiseaseChina, United States, France, Austria, Germany, Italy, Japan, Netherlands, United Kingdom
-
University of MinnesotaRecruitingInflammation | Oxidative Stress | Gaucher Disease Type IUnited States
-
National Institute of Neurological Disorders and...Completed
-
Ultragenyx Pharmaceutical IncCompletedGSD1Netherlands, United States, Canada, Spain