Search clinical trials for: Glycogen Storage Disorders
Total 146121 results
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Manchester University NHS Foundation TrustNorthern Care Alliance NHS Foundation TrustActive, not recruiting
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Duke UniversityCompleted
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Dr. Anupam SehgalTaysha Gene Therapies, Inc.; GlycoNetActive, not recruitingInfantile GM2 Gangliosidosis (Disorder)Canada
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Amicus TherapeuticsCompletedFabry DiseaseJapan, Taiwan, Turkey, United States, Brazil
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Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseUnited States, Netherlands, Australia, Hungary, United Kingdom, Canada, Czechia, Finland, France, Italy, Norway, Slovenia, Spain
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Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseBelgium, United States, United Kingdom, Italy, Czechia, Denmark, Norway
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PfizerCompleted
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Talaris Therapeutics Inc.Duke UniversityTerminatedNiemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...United States
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Chiesi Farmaceutici S.p.A.Chiesi USA, Inc.AvailableFabry DiseaseUnited States
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Massachusetts General HospitalCompleted
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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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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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Chiesi Farmaceutici S.p.A.Iqvia Pty Ltd; ProtalixCompletedFabry DiseaseUnited States, Belgium, Denmark, Italy, United Kingdom
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Lundquist Institute for Biomedical Innovation at...Active, not recruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VIUnited States
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HaEmek Medical Center, IsraelTerminatedLiver Fibrosis | Gaucher Disease | Spleen; FibrosisIsrael
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Mandos LLCCompletedNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Germany, France, New Zealand, Singapore, Spain, Turkey
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Shaare Zedek Medical CenterCompletedGaucher Disease, Type 1Israel
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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 RostockWithdrawnSplenomegaly | Hepatomegaly | Ceramidase Deficiency | Farber's LipogranulomatosisGermany, India, Egypt
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Rigshospitalet, DenmarkUniversity College, LondonCompletedMcArdle DiseaseDenmark, United Kingdom
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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 RostockWithdrawnCholesterol Ester Storage Disease | Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Wolman TypeGermany, India, Sri Lanka
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Nicoline LøkkenCompleted
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Rigshospitalet, DenmarkCompleted
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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 RostockTerminatedMucopolysaccharidosis II | Hunter Syndrome | Hunter's Syndrome, Mild Form | Hunter's Canal SyndromeMexico
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Children's Hospital Medical Center, CincinnatiOhio Injury Prevention Partnership; Whitney Strong OrganizationCompletedMental Health Issue | Suicide and Self-harm | Pediatric Emergency Medicine | Firearm InjuryUnited States
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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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Ultragenyx Pharmaceutical IncCompletedGSD1Netherlands, United States, Canada, Spain
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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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REGENXBIO Inc.Withdrawn
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SanofiApproved for marketing
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National Taiwan University HospitalSanofiCompleted
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Ultragenyx Pharmaceutical IncCompletedGlycogen Storage Disease Type IIIUnited States
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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