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Clinical Trials on Myasthenic Syndromes, Congenital
Total 40721 results
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Palvella Therapeutics, Inc.RecruitingBCCs in Gorlin Syndrome PatientsUnited States, United Kingdom
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Children's Hospital of PhiladelphiaCompletedObstructive Sleep Apnea Syndrome | Down SyndromeUnited States
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National Institute of Allergy and Infectious Diseases...TerminatedEctodermal Dysplasia | Hyper-IgM SyndromeUnited States
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CelgeneRecruitingBeta-thalassemia | Myelodysplastic Syndromes (MDS) | Myeloproliferative Neoplasm(MPN)-Associated MyelofibrosisUnited States, Japan, United Kingdom, Turkey, Greece, France, Australia, Belgium, Bulgaria, Canada, Germany, Israel, Italy, Malaysia, Spain, Sweden, Taiwan, Thailand, Tunisia, Lebanon, China, Netherlands
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Children's Healthcare of AtlantaTerminatedCongenital DisordersUnited States
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The University of QueenslandRecruitingPulmonary Fibrosis | Dyskeratosis Congenita | Telomere Shortening | Telomere DiseaseAustralia
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Maxima Medical CenterRecruitingChronic Pain Syndrome | Diagnosis | Nerve Entrapment Syndrome | Anterior Cutaneous Nerve Entrapment SyndromeNetherlands
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Fondazione TelethonUnknownUsher Syndrome, Type 1BItaly, Netherlands, Spain
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University Medical Centre LjubljanaSlovenian Research AgencyRecruitingPeripheral Nervous System Diseases | Nerve Compression Syndromes | Cubital Tunnel SyndromeSlovenia
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaboratorsRecruitingCongenital Amegakaryocytic Thrombocytopenia | Paroxysmal Nocturnal Hemoglobinuria | Shwachman-Diamond Syndrome | Bone Marrow Failure Syndrome | Congenital Pure Red Cell Aplasia | Hereditary Sideroblastic Anemia | Hematologic Neoplasm With Germline GATA2 Mutation | Hematologic Neoplasm With Germline... and other conditionsUnited States
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ProQR TherapeuticsCompletedEye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf BlindUnited States, Canada, France
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Boston Children's HospitalChildren's Hospital Medical Center, CincinnatiRecruitingShwachman-Diamond Syndrome | Shwachman-Diamond Syndrome-LikeUnited States
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Enzyvant Therapeutics GmBHNational Institute of Allergy and Infectious Diseases (NIAID); Eunice Kennedy... and other collaboratorsCompletedDiGeorge Syndrome | Hypoparathyroidism | Complete DiGeorge SyndromeUnited States
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Mirum Pharmaceuticals, Inc.RecruitingAlagille SyndromeUnited States
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AlbireoCompletedAlagille SyndromeUnited States, United Kingdom, France, Germany, Canada, Netherlands, Italy, Malaysia, Belgium, Israel, New Zealand, Poland, Turkey
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National Human Genome Research Institute (NHGRI)Completed
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National Eye Institute (NEI)Completed
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Neurotech PharmaceuticalsUniversity of California, San FranciscoCompletedRetinitis Pigmentosa | Usher Syndrome Type 2 | Usher Syndrome Type 3United States
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Hôpital le VinatierRecruiting
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Merck Sharp & Dohme LLCActive, not recruitingPIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)United States, Australia, Brazil, Italy, United Kingdom
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ArQule, Inc. (a wholly owned subsidiary of Merck...Worldwide Clinical TrialsTerminatedPIK3CA-Related Overgrowth Spectrum (PROS)/Proteus SyndromeUnited States, Australia, Italy, Spain
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University of Geneva, SwitzerlandCompleted22q11.2 Deletion SyndromeSwitzerland
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Hôpital le VinatierTerminated
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AkesoRecruitingHigher-risk Myelodysplastic SyndromesUnited States, China
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Nuwacell Biotechnologies Co., Ltd.The First Affiliated Hospital of University of Science and Technology of...Not yet recruitingRelapsed / Refractory Myelodysplastic Syndromes
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Wake Forest University Health SciencesNational Cancer Institute (NCI)TerminatedPreviously Treated Myelodysplastic SyndromesUnited States
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ProQR TherapeuticsActive, not recruitingEye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf BlindUnited States, Netherlands, United Kingdom, Germany
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Gilead SciencesCompletedHIV Infections | Renal Impairment | Fanconi Syndrome | Kidney DiseaseCanada, United States
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Stanford UniversityThe Marfan FoundationActive, not recruitingChronic Pain | Marfan Syndrome | Vascular Ehlers-Danlos Syndrome | Loeys-Dietz SyndromeUnited States
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Metcela Inc.RecruitingHypoplastic Left Heart Syndrome | Single VentricleJapan
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Rigel PharmaceuticalsRecruitingLow Risk Myelodysplastic SyndromesUnited States
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Bristol-Myers SquibbActive, not recruitingLower-risk Myelodysplastic SyndromesJapan
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Duke UniversityNational Institute of Mental Health (NIMH); University of Pittsburgh; Harvard...Completed22q11.2 Deletion Syndrome | Velo-Cardio-Facial SyndromeUnited States
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University of Colorado, DenverActive, not recruitingHigh Grade Myelodysplastic SyndromesUnited States
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ALX Oncology Inc.Active, not recruitingHigher Risk Myelodysplastic SyndromesKorea, Republic of, United States, Spain
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Seattle Children's HospitalActelionWithdrawnPulmonary Hypertension | Acute Respiratory Distress Syndrome | Congenital Heart Defects | Neonatal Hypoxic Respiratory Failure | Persistent Pulmonary Hypertension of Newborn
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Boston Children's HospitalCompleted
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Masonic Cancer Center, University of MinnesotaCompletedFanconi AnemiaUnited States
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Hospital Universitari Vall d'Hebron Research InstituteCIEMAT; CIBERERCompleted
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National Heart, Lung, and Blood Institute (NHLBI)Children's Hospital Medical Center, CincinnatiCompletedFanconi AnemiaUnited States
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Baylor College of MedicineCenter for Cell and Gene Therapy, Baylor College of Medicine; University of...TerminatedFANCONI ANEMIAUnited States
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University of MinnesotaCompleted
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Albert Einstein College of MedicineNational Heart, Lung, and Blood Institute (NHLBI)RecruitingDiGeorge Syndrome | 22q11.2 Deletion SyndromeUnited States
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University of Geneva, SwitzerlandTerminated22q11.2 Deletion SyndromeSwitzerland
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Pontificia Universidade Catolica de Sao PauloCompletedEar Malformations in the Velocardiofacial Syndrome
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Rush University Medical CenterEmory UniversityEnrolling by invitationTuberous Sclerosis | Fragile X Syndrome | Rett Syndrome | Hamartoma Syndrome, Multiple | Angelman Syndrome | Creatine Deficiency, X-linked | Telomeric 22Q13 Monosomy Syndrome | Chromosome 15Q, Partial DeletionUnited States
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ProQR TherapeuticsTerminatedEye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf BlindUnited States, United Kingdom
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Emory UniversityCompleted
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Debra Weese-MayerNorthwestern University Feinberg School of Medicine; The Chicago Community...RecruitingCongenital Central Hypoventilation SyndromeUnited States
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Masonic Cancer Center, University of MinnesotaCompleted