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Search clinical trials for: Wolman Disease (early onset LAL Deficiency)
Total 381653 results
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Alexion PharmaceuticalsTerminated
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OrthoCarolina Research Institute, Inc.Enrolling by invitationACL Injury | ACL Tear | ACL - Anterior Cruciate Ligament DeficiencyUnited States
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Lei LiRecruitingEpithelial Ovarian Cancer | Homologous Recombination Deficiency | Homologous Recombination Repair Gene Mutation | Drug Resistance | Overall Survival | Progression-free Survival | Poly(ADP-ribose) Polymerase InhibitorChina
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AstraZenecaActive, not recruitingSolid TumorsBelgium, United Kingdom, Switzerland, Australia
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University Hospital, CaenINSERM U1075 COMETE, UNICAEN, Caen FranceCompletedMuscle Weakness | ACL - Anterior Cruciate Ligament DeficiencyFrance
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Alexion PharmaceuticalsTerminatedLysosomal Acid Lipase DeficiencyUnited Kingdom, Finland, United States, Italy
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West China HospitalWest China Second University HospitalCompletedIron Deficiency Anemia of PregnancyChina
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Chang Gung Memorial HospitalUnknownACL Injury | ACL - Anterior Cruciate Ligament Rupture | ACL - Anterior Cruciate Ligament DeficiencyTaiwan
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iECURE, Inc.RecruitingUrea Cycle Disorders, Inborn | Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency Disease | Ornithine Carbamoyltransferase Deficiency (Disorder)United Kingdom
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Ankara UniversityAlexion PharmaceuticalsCompleted
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Blueprint Medicines CorporationCompletedHepatocellular Carcinoma (HCC)China, Korea, Republic of, Taiwan, Hong Kong, United Kingdom, United States, France, Spain, Switzerland, Singapore, Germany, Italy
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Steadman Philippon Research InstituteÖssur EhfWithdrawnACL | ACL Injury | ACL - Anterior Cruciate Ligament Rupture | ACL - Anterior Cruciate Ligament DeficiencyUnited States
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Origin BiosciencesCompletedMolybdenum Cofactor Deficiency (MoCD) | Rare Autosomal Recessive Disorder | Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)United States
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Institute of Liver and Biliary Sciences, IndiaRecruiting
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Swiss Federal Institute of TechnologyMahidol UniversityCompletedIron Deficiency | Vitamin A Deficiency | Zinc DeficiencySwitzerland, Thailand
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Inozyme PharmaEngage Health Inc.; GACI GlobalCompletedGeneralized Arterial Calcification in Infancy | Autosomal Recessive Hypophosphatemic Rickets Type 2United States
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Alexion PharmaceuticalsCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited Kingdom, France, United States, Egypt, Ireland
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University of MinnesotaSanofi; Moran, Antoinette, M.D.Completed
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National Institute of Allergy and Infectious Diseases...CompletedChronic Granulomatous DiseaseUnited States
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Hui BuUnknownCryptococcal Meningitis
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Inozyme PharmaNot yet recruitingGeneralized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency | ATP-Binding Cassette Subfamily C Member 6 Deficiency
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University of PennsylvaniaTerminatedOrnithine Transcarbamylase Deficiency Disease
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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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Eunice Kennedy Shriver National Institute of Child...Children's National Research InstituteTerminatedPhase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase Deficiency Disease
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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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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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Wageningen UniversityCCS Haryana Agricultural University Hisar, IndiaCompleted
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University of Colorado, DenverCompletedIron Deficiency Anemia | Iron DeficiencyUnited States
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University of Colorado, DenverColorado Clinical & Translational Sciences InstituteRecruiting
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Children's Hospital of PhiladelphiaLucky Iron FishCompletedIron-deficiency | Iron Deficiency AnemiaDominican Republic
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Emory UniversityCenters for Disease Control and PreventionCompleted
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Mostafa Hassanein, MscCompletedACL Injury | Knee Deformity | ACL - Anterior Cruciate Ligament Deficiency | Varus Deformity of Tibia (Disorder)Egypt
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Rare Thyroid Therapeutics International ABVitaccess LtdCompletedAllan-Herndon-Dudley Syndrome | Monocarboxylate Transporter 8 (MCT8) DeficiencyUnited Kingdom
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Shanghai Hutchison Pharmaceuticals LimitedNot yet recruitingCervical Spondylotic Myelopathy
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rEVO BiologicsCompletedAntithrombin Deficiency, CongenitalFrance, United Kingdom, Germany, United States, Sweden, Italy
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Children's National Research InstituteNational Center for Advancing Translational Sciences (NCATS)RecruitingHypoxic-Ischemic Encephalopathy | Urea Cycle Disorder | Organic Acidemia | Fatty Acid Oxidation Disorder | Maple Syrup Urine Disease | Glutaric Acidemia IUnited States
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Amsterdam Molecular TherapeuticsThe Clinical Trial CompanyCompletedFamilial Lipoprotein Lipase DeficiencyCanada
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Massachusetts General HospitalAlexion PharmaceuticalsUnknownCholesterol Ester Storage Disease | Lysosomal Acid Lipase DeficiencyUnited States
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Universitair Ziekenhuis BrusselNot yet recruiting
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National Heart, Lung, and Blood Institute (NHLBI)CompletedLung Diseases | Chronic Obstructive Pulmonary Disease | Emphysema | Alpha-1 Antitrypsin Deficiency
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Great Ormond Street Hospital for Children NHS Foundation...Completed
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GlaxoSmithKlineCompletedColorectal CancerUnited States, Canada
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AEterna ZentarisNovo Nordisk A/SActive, not recruitingGrowth Hormone DeficiencyUnited States, Italy, Turkey, Georgia, Romania, Poland, Armenia, Germany, Serbia, Slovakia, Slovenia
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University of Alabama at BirminghamGlaxoSmithKlineTerminatedOvarian CancerUnited States
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Baxalta now part of ShireCompletedHemophilia A | Hemophilia BUnited States
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Adiyaman UniversityCompletedVitamin D Deficiency | Optic Nerve Diseases | Pediatric Disorder | Retinal Disease | Choroidal DiseaseTurkey
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Rare Thyroid Therapeutics International ABPremier Research Group plc; Egetis TherapeuticsRecruitingMonocarboxylate Transporter 8 Deficiency | Allan-Herndon-Dudley SyndromeUnited States, United Kingdom, Netherlands
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Rhythm Pharmaceuticals, Inc.CompletedObesity | Genetic Obesity | Obesity Due to Melanocortin 4 Receptor DeficiencyUnited States, United Kingdom, Germany, Netherlands, France, Canada, Greece, Israel, Spain
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Instituto Nacional de Salud Publica, MexicoCompletedInflammation | Anemia | Nutritional Deficiency