Clinical Trials on Brain Diseases, Metabolic, Inborn
Total 87583 results
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University of FloridaBioMarin PharmaceuticalCompleted
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LMU KlinikumGenzyme, a Sanofi CompanyCompletedPompe DiseaseGermany, Taiwan, Italy
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Amicus TherapeuticsCompleted
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National Taiwan University HospitalCompleted
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National Taiwan University HospitalUnknown
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type IIUnited States, France, Germany, Israel, Italy, Netherlands, Taiwan
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Fundació Institut de Recerca de l'Hospital de la...Genzyme, a Sanofi CompanyUnknown
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National Taiwan University HospitalCompleted
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Amicus TherapeuticsCompletedGlycogen Storage Disease Type II | Pompe DiseaseUnited States
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BioMarin PharmaceuticalCompletedHyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) DeficiencySweden, France, Italy, Spain, Germany, Netherlands, Austria, Slovakia, Portugal
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Travere Therapeutics, Inc.RecruitingNatural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)Homocystinuria Due to CBS DeficiencyIreland, Qatar, United States, United Kingdom
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National Center for Research Resources (NCRR)University of California, San DiegoCompletedHomocystinuria
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University of Massachusetts, AmherstWithdrawnOxidative Stress | COPD | Tetrahydrobiopterin DeficiencyUnited States
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Amicus TherapeuticsRecruitingPompe DiseaseUnited States, Germany
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Duke UniversitySanofiRecruiting
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Duke UniversityGenzyme, a Sanofi CompanyRecruitingPompe DiseaseUnited States
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Genzyme, a Sanofi CompanyCompleted
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Valerion Therapeutics, LLCTerminatedPompe DiseaseUnited States, United Kingdom
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University of FloridaNational Heart, Lung, and Blood Institute (NHLBI)CompletedPompe DiseaseUnited States
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BioMarin PharmaceuticalCompletedPompe DiseaseUnited States, United Kingdom, France, Australia, Germany
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BioMarin PharmaceuticalTerminatedPompe DiseaseUnited States, United Kingdom, Australia, France, Germany, New Zealand
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University of FloridaCompletedPompe DiseaseUnited States
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National Taiwan University HospitalUnknown
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Genzyme, a Sanofi CompanyTerminatedPompe DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Pompe DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type IIUnited States, Taiwan, Israel, United Kingdom, France
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Assistance Publique - Hôpitaux de ParisNot yet recruiting
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Freeline TherapeuticsRecruitingGaucher Disease, Type 1Spain, Israel, United States, United Kingdom, Brazil, Germany, Paraguay
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Massachusetts General HospitalRecruiting
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CENTOGENE GmbH RostockWithdrawnSkeletal Abnormalities | Psychomotor RetardationGermany, India, Sri Lanka, Egypt
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Genzyme, a Sanofi CompanyActive, not recruiting
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Centre Hospitalier de CayenneCompleted
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Ultragenyx Pharmaceutical IncActive, not recruitingOrnithine Transcarbamylase (OTC) DeficiencyCanada, United States, France, Spain, United Kingdom
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Lysosomal and Rare Disorders Research and Treatment...RecruitingBone Diseases | Gaucher DiseaseUnited States
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Assiut UniversityNot yet recruiting
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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
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SanofiCompletedGaucher Disease | SplenomegalyFrance
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The Hospital for Sick ChildrenActelionCompletedGangliosidoses GM2Canada
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Rambam Health Care CampusUnknown
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Massachusetts General HospitalActive, not recruiting
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SanofiCompletedGaucher DiseaseUnited States
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IntraBio IncCompletedGM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Germany, Spain, United Kingdom
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Terence FlotteMassachusetts General Hospital; University of Massachusetts, WorcesterActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States
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National Institute of Neurological Disorders and...CompletedGaucher DiseaseUnited States, United Kingdom
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Andrea GropmanChildren's National Research InstituteCompletedOrnithine Transcarbamylase Deficiency
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Travere Therapeutics, Inc.Recruiting
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Hospital de Clinicas de Porto AlegreFundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, BrazilSuspendedCBS DeficiencyBrazil
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Serhat Gumrukcu, MD PhDFrida Therapeutics LLCRecruiting
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Amicus TherapeuticsAvailable