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Clinical Trials on Neutral Lipid Storage Disease With Myopathy (NLSD-M)
Total 511 results
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ShireCompletedMetachromatic Leukodystrophy (MLD)Australia, Denmark, Japan, France, Germany
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ShireTerminatedLate Infantile Metachromatic LeukodystrophyDenmark
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TakedaWithdrawnMetachromatic Leukodystrophy (MLD)Spain
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ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)United States, Canada, Belgium, Israel, Netherlands, United Kingdom, Germany, Spain, Japan, Brazil, Argentina, France, Greece, Italy
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ShireCompletedMetachromatic Leukodystrophy (MLD)Denmark
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ShireCompletedLate Infantile Metachromatic LeukodystrophyDenmark
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Assistance Publique - Hôpitaux de ParisEuropean Leukodystrophy AssociationCompletedLate Infantile Metachromatic LeukodystrophyFrance
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University of California, San FranciscoDuke UniversityRecruitingWolman Disease | MPS IVA | Pompe Disease Infantile-Onset | Gaucher Disease, Type 2 | MPS VI | MPS I | Gaucher Disease, Type 3 | MPS II | Mps VIIUnited States
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SanofiRecruitingGaucher's Disease Type IIIUnited States, Germany, Hungary, France, China, Japan, Canada, Argentina, Italy, Turkey
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ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)Denmark, Germany, Japan, Australia, France, Brazil, Czechia
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National Organization for Rare DisordersFood and Drug Administration (FDA)Active, not recruitingMetachromatic LeukodystrophyUnited States
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University of PittsburghActive, not recruitingMetachromatic LeukodystrophyUnited States
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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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Shenzhen Geno-Immune Medical InstituteUnknown
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGaucher Disease Type 1United States
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Children's National Research InstituteActelionCompletedSandhoff Disease | GM2 Gangliosidoses | Tay-SachsUnited States
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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
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Azafaros A.G.Active, not recruitingGM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Brazil, France, Germany, Italy, United Kingdom
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University of MinnesotaRecruitingInflammation | Oxidative Stress | Gaucher Disease Type IUnited States
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National Institute of Neurological Disorders and...Completed
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Dr. Anupam SehgalTaysha Gene Therapies, Inc.; GlycoNetActive, not recruitingInfantile GM2 Gangliosidosis (Disorder)Canada
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Rabin Medical CenterGenzyme, a Sanofi CompanyUnknown
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PfizerNo longer availableGaucher DiseaseUnited States, Israel
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Zymenex A/SCompletedInfantile Globoid Cell LeukodystrophyUnited States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedTay Sachs Disease | Sandhoff Disease | Late Onset Tay Sachs DiseaseUnited States
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SanofiActive, not recruitingGaucher's Disease Type III | Gaucher's Disease Type ICanada, Argentina, France, Italy, Japan, Russian Federation, Spain, Sweden, Turkey, United Kingdom
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Children's Hospital Medical Center, CincinnatiCompletedOsteopenia | Gaucher's DiseaseIsrael, United States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsTerminatedTay-Sachs Disease | Sandhoff Disease | GM1 Gangliosidoses | GM2 GangliosidosesUnited States
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National Human Genome Research Institute (NHGRI)Sio Gene TherapiesRecruitingGangliosidosis | Lysosomal Diseases | GM1United States
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University of California, Los AngelesRecruitingDegenerative Disc Disease | Cervical Radiculopathy | Cervical Disc Herniation | Cervical Spondylosis With MyelopathyUnited States
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CENTOGENE GmbH RostockWithdrawnSplenomegaly | HepatomegalyGermany, India, Sri Lanka
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M.A.G.I.C. Clinic LTDHanalytics Solutions Inc.RecruitingMitochondrial Diseases | Fabry Disease | Metabolic Disease | Gaucher Disease | Pompe DiseaseCanada
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Genzyme, a Sanofi CompanyCRL/MedinetTerminatedFabry Disease | Chronic Kidney Disease, Stage IV (Severe)United States
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Hospices Civils de LyonUnknownEnd Stage Renal Disease | Fabry Disease | Renal DialysisFrance
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BioMarin PharmaceuticalTerminatedLate-onset Pompe Patients Untreated or Treated With rhGAASpain, Slovenia, Serbia, Korea, Republic of, Greece, Australia, Singapore, Brazil, Canada, Taiwan, Ireland, Poland, Romania
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Exsar CorporationThe Hospital for Sick Children; NYU Langone Health; University Hospitals Cleveland...WithdrawnG(M2) Ganglioside | Tay-Sachs Disease Ganglioside | Sandhoff Disease GangliosideUnited States, Canada
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National Human Genome Research Institute (NHGRI)CompletedParkinson Disease | Gaucher DiseaseUnited States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Late Onset Tay-Sachs DiseaseUnited States
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Lysosomal and Rare Disorders Research and Treatment...UnknownParkinson Disease | Gaucher DiseaseUnited States
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Groupe Hospitalier Diaconesses Croix Saint-SimonTerminated
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Massachusetts General HospitalCompleted
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University Hospital, RouenUnknownAnderson-Fabry DiseaseFrance
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SanofiRecruitingFabry's DiseaseUnited States
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Hospital Clinico Universitario de SantiagoAlvaro Hermida (Co-IP); Susana Belen Bravo; Maria Teresa Cardoso; Cristobal Colon... and other collaboratorsRecruiting
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Amicus Therapeutics France SASActive, not recruitingFabry Disease | Anderson Fabry DiseaseFrance
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National Institute of Neurological Disorders and...Completed
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Chiesi Farmaceutici S.p.A.ICON plcNot yet recruiting
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UniQure Biopharma B.V.Not yet recruiting
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Brunel UniversityRoyal Free Hospital NHS Foundation TrustRecruiting