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Search clinical trials for: Lysosomal Storage Diseases, Nervous System
Total 248327 results
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CENTOGENE GmbH RostockWithdrawnSkeletal Abnormalities | Psychomotor RetardationGermany, India, Sri Lanka, Egypt
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CENTOGENE GmbH RostockWithdrawnNiemann-Pick Disease, Type C | Niemann-Pick DiseaseGermany, India, Sri Lanka
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CENTOGENE GmbH RostockWithdrawnSeizures | Mental Retardation | Hepato-splenomegaly | Dysostosis MultiplexEgypt, Germany, India, Sri Lanka
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CENTOGENE GmbH RostockWithdrawnSplenomegaly | HepatomegalyGermany, India, Sri Lanka
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CENTOGENE GmbH RostockWithdrawnChronic Kidney Disease | Hearing Loss | Angiokeratomas | Ocular AbnormalitiesSri Lanka, India, Germany
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CENTOGENE GmbH RostockWithdrawnFructose Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease | Glycogen Storage Disease Type V | Glycogen Storage Disease Type I | Glycogen Storage Disease Type III | Glycogen Storage Disease Type VII | Glycogen Storage Disease Type IV | Glycogen Storage Disease Type... and other conditionsGermany, India, Sri Lanka
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CENTOGENE GmbH RostockWithdrawnSplenomegaly | Hepatomegaly | Ceramidase Deficiency | Farber's LipogranulomatosisGermany, India, Egypt
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CENTOGENE GmbH RostockWithdrawnKrabbe DiseaseEgypt, Germany, India, Sri Lanka
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CENTOGENE GmbH RostockWithdrawnMuscle Weakness | Peripheral NeuropathyEgypt, Germany, India, Sri Lanka
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CENTOGENE GmbH RostockWithdrawnLung Diseases | Eye Abnormalities | Lysosomal Storage Disease | Macroglossia | Obstructive Sleep ApnoeaGermany, India, Sri Lanka, Egypt
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CENTOGENE GmbH RostockWithdrawnHypophosphatemia | Photophobia | Renal Fanconi SyndromeGermany, India, Sri Lanka
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CENTOGENE GmbH RostockWithdrawnLung Disease | Cerebral Aneurysm | Macroglossia | Cardiac Diseases | Obstructive Sleep Apnoea | Muscular Weakness | Hepato-splenomegalyGermany, India, Sri Lanka
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CENTOGENE GmbH RostockTerminatedMucopolysaccharidosis II | Hunter Syndrome | Hunter's Syndrome, Mild Form | Hunter's Canal SyndromeMexico
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KemPharm Denmark A/STerminatedGaucher Disease, Type 1 | Gaucher Disease, Type 3India
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BioMarin PharmaceuticalTerminatedLate-onset Pompe DiseaseUnited States, United Kingdom
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JCR Pharmaceuticals Co., Ltd.Completed
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JCR Pharmaceuticals Co., Ltd.Completed
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JCR Pharmaceuticals Co., Ltd.Completed
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Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Disease | Metachromatic LeukodystrophyItaly
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Shaare Zedek Medical CenterShireCompletedGaucher Disease, Type 1
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Shaare Zedek Medical CenterPfizerCompletedGaucher Disease, Type 1
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Sangamo TherapeuticsTerminatedMucopolysaccharidosis II | MPS IIUnited States
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New York Medical CollegeCompletedMucopolysaccharidosis I | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Fucosidosis | Gaucher... and other conditionsUnited States
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Turkish Society of Pediatric Gastroenterology,...CompletedDuchenne Muscular Dystrophy | Pompe Disease (Late-onset)Turkey
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REGENXBIO Inc.Withdrawn
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SanofiApproved for marketing
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National Taiwan University HospitalSanofiCompleted
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Assistance Publique - Hôpitaux de ParisShire International GmbHCompleted
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Eunice Kennedy Shriver National Institute of Child...TerminatedNiemann-Pick Disease, Type C1United States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Pompe DiseaseUnited States
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LMU KlinikumCompletedHealthy | Myotonic Dystrophy Type 2 | Pompe Disease (Late-onset) | Spinal Muscular Atrophy Type 3 | Myotonic Dystrophy Type 1 (DM1) | Facioscapulohumeral Muscular Dystrophy 1 | Inclusion Body Myositis, SporadicGermany
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Duke UniversityWithdrawnPompe Disease (Late-onset)United States
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Idorsia Pharmaceuticals Ltd.CompletedFabry DiseaseUnited States, Spain, Belgium, Australia, Austria, Canada, Germany, Ireland, Italy, Netherlands, Norway, Poland, Switzerland, United Kingdom
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Ari ZimranPfizerActive, not recruitingGaucher Disease, Type 3Israel, Turkey, India
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Talaris Therapeutics Inc.Duke UniversityNo longer availableMetachromatic LeukodystrophyUnited States
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Genzyme, a Sanofi CompanyCompletedGaucher DiseaseCanada, Russian Federation, Tunisia
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CENTOGENE GmbH RostockTerminatedDeafness | Immunodeficiency | Mental Retardation | Gingival Hypertrophy | Skeletal Abnormalities | Facial DysmorphismGermany, Switzerland
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University of UtahGenzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States
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ActelionCompleted
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TakedaCompletedMucopolysaccharidosis (MPS)Brazil
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REGENXBIO Inc.CompletedMucopolysaccharidosis IIBrazil, United States, United Kingdom
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SanofiCompletedGaucher Disease | SplenomegalyFrance
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type IIChina
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Genzyme, a Sanofi CompanyTerminatedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease | Glycogenesis 2 Acid Maltase DeficiencyUnited States, Israel
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University of FloridaLacerta TherapeuticsCompletedPompe DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedPompe Disease | Glycogen Storage Disease Type II (GSD II)United States, Russian Federation, United Kingdom, Bulgaria, India, Ukraine
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Genzyme, a Sanofi CompanyCompletedSphingomyelin LipidosisBrazil, France, Italy, United States, Germany, United Kingdom
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ShireCompletedGaucher DiseaseUnited States, Spain, United Kingdom, Israel
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Institut National de la Santé Et de la Recherche...Assistance Publique - Hôpitaux de Paris; European Leukodystrophy AssociationActive, not recruitingMetachromatic LeukodystrophyFrance
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University Hospital, ToursCompletedMucopolysaccharidosesFrance