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Clinical Trials on Leaky SCID in Germany
Total 100 results
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Nutricia ResearchCompletedPhenylketonuriasGermany, Netherlands
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Recordati Rare DiseasesRecruitingPropionic Acidemia | Methylmalonic AcidemiaFrance, Spain, Italy, United Kingdom, Norway, Germany, Sweden
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Dr. Loges & Co. GmbHBioTeSys GmbHCompletedEndothelial Dysfunction | Hyperhomocysteinemia | Hypertension Grade I, Subgroup "Borderline" (WHO)Germany
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NestléCompletedPhenylketonuriaAustralia, Germany
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DLR German Aerospace CenterUniversity of Aarhus; University of Trieste; European Space AgencyCompletedSprains and Strains | Body Weight Changes | Muscular Atrophy | Bone Diseases, Metabolic | Cardiovascular Abnormalities | Acid-Base Imbalance | Protein MetabolismGermany
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DLR German Aerospace CenterUniversity Hospital, Angers; University of Zurich; University Hospital, Bonn; European... and other collaboratorsCompletedSprains and Strains | Body Weight Changes | Muscular Atrophy | Bone Diseases, Metabolic | Cardiovascular Abnormalities | Acid-Base Imbalance | Protein MetabolismGermany
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PTC TherapeuticsGenzyme, a Sanofi CompanyTerminatedAmino Acid Metabolism, Inborn ErrorsFrance, United Kingdom, Italy, Belgium, Germany, Switzerland
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SangartCompletedShock, Hemorrhagic | Acidosis, Lactic | Shock, TraumaticFrance, United Kingdom, South Africa, Germany
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Cytonet GmbH & Co. KGCompletedOrnithine Transcarbamylase Deficiency | Urea Cycle Disorders | Citrullinemia | Carbamoylphosphate Synthetase I DeficiencyGermany
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Johann Wolfgang Goethe University HospitalMedical University of Graz; University Hospital, Angers; Medical University of... and other collaboratorsCompletedIron-deficiency | Anemia | Vitamin B 12 Deficiency | Folic Acid Deficiency | Anaemia of Renal Disease | Anemia of Chronic InflammationGermany
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CENTOGENE GmbH RostockWithdrawnMetabolic Disorders | Tyrosinosis | Hepatorenal Tyrosinemia | Fumarylacetoacetase Deficiency | Fah DeficiencyGermany, Sri Lanka, India
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Ludwig-Maximilians - University of MunichEuropean UnionCompletedPhenylketonuriaGermany, Italy, Spain, United Kingdom
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Technische Universität DresdenGWT-TUD GmbHCompletedPrimary Immunodeficiency (PID) | Secondary Immunodeficiency (SID) | Neurological Autoimmune DiseaseGermany
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Swedish Orphan BiovitrumCompletedEfficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 (HT-1)Hereditary Tyrosinemia, Type IBelgium, Denmark, France, Germany, Sweden
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Swedish Orphan BiovitrumCompletedHereditary Tyrosinemia, Type IGermany, United Kingdom, France
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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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Cytonet GmbH & Co. KGCRS Clinical Research Services Mannheim GmbHCompleted
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BioMarin PharmaceuticalSyneos HealthRecruitingPhenylketonuria, MaternalGermany, United States, Italy
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BioMarin PharmaceuticalCompletedPhenylketonuriaItaly, Spain, United Kingdom, Germany
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LMU KlinikumSeventh Framework Programme; NBIA AllianceRecruitingNeurodegeneration With Brain Iron Accumulation (NBIA) | Pantothenate Kinase-associated Neurodegeneration (PKAN) | Aceruloplasminemia | Beta-Propeller Protein-Associated Neurodegeneration (BPAN) | Mitochondrial Membrane Protein Associated Neurodegeneration (MPAN) | Fatty Acid Hydroxylase-associated... and other conditionsCanada, Czechia, Germany, Italy, Netherlands, Poland, Serbia, Spain
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SangartCompletedShock, Hemorrhagic | Acidosis, Lactic | Shock, TraumaticNorway, Singapore, Israel, France, United Kingdom, Germany, Switzerland, Australia, Brazil, South Africa, New Zealand, Colombia, Austria, Spain
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SangartWithdrawnTrauma | Hemorrhagic Shock | Lactic AcidosisNew Zealand, United Kingdom, Israel, France, Switzerland, Australia, Belgium, Brazil, Germany, Norway, South Africa
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BioMarin PharmaceuticalRecruitingPhenylketonuria (PKU)United States, Germany, Italy
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BioMarin PharmaceuticalCompletedTetrahydrobiopterin Deficiencies | Hyperphenylalaninemia, Non-PhenylketonuricGermany, United States
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BioMarin PharmaceuticalCompletedPhenylketonuriaItaly, Turkey, United Kingdom, Germany, Netherlands, Slovakia, Austria, Belgium, Czechia
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Origin BiosciencesCompletedMolybdenum Cofactor Deficiency | Isolated Sulfite Oxidase DeficiencySpain, Israel, United States, United Kingdom, Canada, Netherlands, Germany, Saudi Arabia, Tunisia, Japan, Italy, Malaysia, Poland, Turkey
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BioMarin PharmaceuticalCompletedPhenylketonuria (PKU)United States, Turkey, Canada, France, Germany, Italy, United Kingdom
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BioMarin PharmaceuticalActive, not recruitingPhenylketonuria (PKU)United States, Germany
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Andrea GropmanEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruitingBrain Diseases, Metabolic, Inborn | Amino Acid Metabolism, Inborn Errors | Urea Cycle DisordersUnited States, Canada, Germany, Switzerland
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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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Kaleido BiosciencesTerminatedUrea Cycle DisorderBelgium, United States, Spain, United Kingdom, Germany, Switzerland, Turkey
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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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Khondrion BVJulius Clinical; ProPharma Group; CertaraCompletedMitochondrial Diseases | Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation | Maternally Inherited Diabetes and Deafness (MIDD) | Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke Like Episodes (MELAS) | Chronic Progressive External Ophthalmoplegia (CPEO)Netherlands, Denmark, United Kingdom, Germany
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PTC TherapeuticsRecruitingPhenylketonuriaUnited States, Germany, Spain, Australia, Brazil, United Kingdom, Denmark, Mexico, Portugal, Turkey, Canada, Georgia, Italy, Netherlands, Japan
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Italy, Canada, France, United Kingdom, Austria, 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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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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Ultragenyx Pharmaceutical IncRecruitingOTC DeficiencyUnited States, Portugal, Italy, Spain, United Kingdom, Japan, Canada, Germany, France, Argentina, Netherlands, Brazil
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PTC TherapeuticsCompletedPhenylketonuriaSpain, United States, Australia, Germany, United Kingdom, Brazil, Denmark, Canada, France, Georgia, Italy, Mexico, Netherlands, Portugal, Turkey
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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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TakedaRecruitingMultiple Myeloma | Secondary Immunodeficiency (SID)Greece, Spain, Italy, Romania, Argentina, Czechia, France, Germany, Poland, Sweden, Turkey
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Swedish Orphan BiovitrumCompletedHereditary Tyrosinemia, Type IBelgium, Denmark, France, Germany, Austria, Czechia, Finland, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom
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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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Enzyvant Therapeutics GmBHCompletedFarber Disease | Farber's Disease | Farber Lipogranulomatosis | Acid Ceramidase Deficiency | Ceramidase Deficiency | N-Laurylsphingosine Deacylase Deficiency | ASAH1 MutationUnited States, Egypt, Canada, Italy, Turkey, Argentina, Germany, India, Sweden
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Novartis PharmaceuticalsActive, not recruitingSickle Cell Disease (SCD)United States, United Kingdom, Germany, Spain, Greece, Italy, Brazil, Lebanon, Turkey, Belgium, France, Jordan, India, Finland, Canada, Colombia, Netherlands, Oman, South Africa, Panama, Ghana
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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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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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Novartis PharmaceuticalsRecruitingSickle Cell Disease (SCD)United States, Germany, Spain, Belgium, Italy, Lebanon, Brazil, Colombia, Oman, Switzerland, Turkey, United Kingdom, Canada, India, France
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Tisento TherapeuticsNot yet recruitingMitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)United States, Australia, Italy, Germany, Canada, United Kingdom
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Rigshospitalet, DenmarkUniversity College, LondonEnrolling by invitationEnd Stage Liver Disease | Cardiovascular Diseases | Fractures, Bone | Diabetes Mellitus | Renal Insufficiency | Hepatitis B | Hepatitis C | Kidney Failure, Chronic | Proteinuria | HIV | Coinfection | Acidosis, Lactic | AIDSSpain, Switzerland, Germany, United Kingdom, Austria, Sweden, Finland, Israel, Denmark, France, Italy, Iceland, Belgium, Slovakia, Norway, Lithuania, Portugal, Poland, Croatia, Czechia, Greece, Hungary, Luxembourg, Argentina, Belarus, Bosnia... and more