Clinical Trials on Centronuclear Myopathy, X-Linked
Total 1549 results
-
Assistance Publique - Hôpitaux de ParisTerminatedNephrolithiasis | Dent's DiseaseFrance
-
Assistance Publique - Hôpitaux de ParisRecruitingAdrenoleukodystrophy | Adrenomyeloneuropathy | Metachromatic Leukodystrophy | Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented GliaFrance
-
Masonic Cancer Center, University of MinnesotaTerminatedSickle Cell Disease | Thalassemia | Cerebral Adrenoleukodystrophy | Inherited Metabolic Disorders | High Risk Hematologic DisordersUnited States
-
Baylor College of MedicineThe Methodist Hospital Research Institute; Center for Cell and Gene Therapy...CompletedAcute Myeloid Leukemia | Acute Lymphoblastic Leukemia | Myelodysplastic Syndrome | Chronic Myelogenous Leukemia | Hemophagocytic Lymphohistiocytosis (HLH) | Non Hodgkins Lymphoma | Familial Hemophagocytic Lymphohistiocytosis (FLH) | Viral-associated Hemophagocytic Syndrome (VAHS) | X-linked Lymphoproliferative...United States
-
Hugo W. Moser Research Institute at Kennedy Krieger...RecruitingNeurodegenerative Diseases | Ataxia | Cerebellar Ataxia | Leukodystrophy | Adrenomyeloneuropathy | LBSL | Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation | Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)United States
-
IRCCS Eugenio MedeaRecruitingMuscular Dystrophies | Becker Muscular Dystrophy | Limb Girdle Muscular Dystrophy | Facio-Scapulo-Humeral DystrophyItaly
-
Vanderbilt University Medical CenterRecruitingDuchenne Muscular DystrophyUnited States
-
National Institute of Allergy and Infectious Diseases...RecruitingChronic Granulomatous Disease | CGDUnited States
-
Region StockholmKarolinska InstitutetRecruitingBipolar Disorder | Bipolar Depression | Bipolar I Disorder | Bipolar II Disorder | Bipolar Affective Disorder; Remission in | Bipolar Affective Disorder, Currently Depressed, ModerateSweden
-
University Hospital OlomoucNot yet recruiting
-
Sanguine BiosciencesTerminatedChronic Granulomatous DiseaseUnited States
-
PTC TherapeuticsCompletedNervous System Diseases | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Muscular Diseases | Neuromuscular Diseases | Muscular Dystrophies | Muscular Disorders, Atrophic | Muscular Dystrophy, Duchenne | Musculoskeletal DiseaseUnited States, Taiwan, India, Korea, Republic of, Thailand, Australia, Brazil, Bulgaria, Canada, China, Hong Kong, Malaysia, Mexico, Poland, Puerto Rico, Russian Federation, Turkey, Japan
-
Arcturus Therapeutics, Inc.CompletedOrnithine Transcarbamylase DeficiencyNew Zealand
-
Freeline TherapeuticsCompletedHemophilia B | Blood Coagulation Disorders, Inherited | Blood Coagulation Disorder | Haematologic DiseaseUnited States, United Kingdom, Australia, Netherlands, Austria, France, Ireland, Italy, Turkey, Canada, Germany, South Africa
-
Istituto Auxologico ItalianoCompletedOsteoporosis | Duchenne Muscular Dystrophy
-
Catalyst BiosciencesTerminatedHemophilia A With Inhibitor | Hemophilia B With InhibitorUnited States, United Kingdom, Taiwan, Italy, Spain, India, Turkey, Malaysia, Russian Federation, Armenia, Georgia, Hungary, Mexico, Poland, South Africa, Ukraine
-
Novo Nordisk A/SCompletedHaemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia BFrance, United States, India, Mexico, United Kingdom, Netherlands, Sweden, Korea, Republic of, Spain, Switzerland, Serbia, Turkey, Bulgaria, Italy, Japan, Poland, South Africa, Croatia, Algeria, Australia, Bosnia and Herzegovina, ... and more
-
Assistance Publique - Hôpitaux de ParisAssociation Française contre les Myopathies (AFM), ParisCompletedHeart Failure | Duchenne Muscular Dystrophy | CardiomyopathyFrance
-
Safaa AA KhaledUnknownHemophilia | Von Willebrand Diseases | Christmas Disease
-
Translate Bio, Inc.WithdrawnOrnithine Transcarbamylase Deficiency
-
University of North Carolina, Chapel HillNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaboratorsCompletedMuscular Dystrophies | Duchenne Muscular Dystrophy | Becker Muscular Dystrophy | Limb-Girdle Muscular DystrophyUnited States
-
National Institute of Allergy and Infectious Diseases...Recruiting
-
Peking Union Medical College HospitalNational Natural Science Foundation of China; Cancer Institute and Hospital...Recruiting
-
Northwestern UniversityCompletedBecker Muscular Dystrophy | Limb-girdle Muscular DystrophyUnited States
-
Jiangsu Gensciences lnc.CompletedFactor VII Deficiency | Hemophilia A With Inhibitor | Hemophilia B With InhibitorChina
-
Hacettepe UniversityCompletedDuchenne Muscular Dystrophy | Ambulation Difficulty | Ambulation Disorder, NeurologicTurkey
-
Groupe Maladies hémorragiques de BretagneCompletedHemophilia A | Hemophilia B | Anticoagulant-induced BleedingFrance
-
Hacettepe UniversityCompletedDuchenne Muscular Dystrophy | Gait Disorders, Neurologic | Gait Disorders in ChildrenTurkey
-
Newcastle-upon-Tyne Hospitals NHS TrustCompletedDuchenne Muscular DystrophyUnited Kingdom
-
Duke UniversityUniversity of FloridaCompleted
-
Ultragenyx Pharmaceutical IncCompletedOrnithine Transcarbamylase DeficiencyUnited States
-
Jett Foundation, Inc.Pfizer; Hoffmann-La Roche; Italfarmaco; Nationwide Children's Hospital; Capricor... and other collaboratorsCompletedDuchenne Muscular Dystrophy | Rare Diseases | Disability Physical | Burden, Dependency | Impairment | Disease ManagementUnited States
-
Washington University School of MedicineCompletedBarth Syndrome
-
Institut National de la Santé Et de la Recherche...RecruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Mucopolysaccharidosis III | Mucopolysaccharidosis IV | Mucopolysaccharidosis VII | Multiple Sulfatase Deficiency Disease | Mucopolysaccharidosis IXFrance
-
Arcturus Therapeutics, Inc.RecruitingOTC Deficiency | Ornithine Transcarbamylase Deficiency | OTCDSpain, Belgium, United Kingdom, Sweden
-
Children's Hospital of Fudan UniversityRecruitingBCG | Vitamin D3 | Chronic-granulomatous DiseaseChina
-
Masonic Cancer Center, University of MinnesotaRecruitingAplastic Anemia | Dyskeratosis CongenitaUnited States
-
Boston Children's HospitalNational Institute of Neurological Disorders and Stroke (NINDS)RecruitingLimb-girdle Muscular Dystrophy | Neuromuscular; Disorder, Hereditary | Duchenne/Becker Muscular DystrophyUnited States
-
National Institute of Allergy and Infectious Diseases...Active, not recruitingChronic Granulomatous Disease TransplantUnited States
-
David WilliamsActive, not recruitingWiskott-Aldrich SyndromeUnited States
-
Stealth BioTherapeutics Inc.CompletedBarth SyndromeUnited States
-
University of PittsburghRespirtech, Inc.CompletedDuchenne Muscular DystrophyUnited States
-
Fondazione TelethonOspedale San RaffaeleActive, not recruitingWiskott-Aldrich SyndromeItaly, United States
-
American Thrombosis and Hemostasis NetworkTakeda; CSL Behring; OctapharmaCompletedHemophilia A | Hemophilia B | Hemophilia | Hemophilia A With Inhibitor | Haemophilia | Hemophilia B With Inhibitor | Haemophilia A Without Inhibitor | Haemophilia B Without InhibitorUnited States
-
Ultragenyx Pharmaceutical IncTerminatedOrnithine Transcarbamylase DeficiencyUnited States
-
National Institute of Allergy and Infectious Diseases...Rare Diseases Clinical Research Network; Primary Immune Deficiency Treatment...Enrolling by invitationGranulomatous Disease, ChronicUnited States, Canada
-
National Institute of Allergy and Infectious Diseases...CompletedAtherosclerosis | Chronic Granulomatous Disease (CGD)United States
-
GenethonInstitute of Child Health; Great Ormond Street Hospital for Children NHS Foundation...Completed
-
GlaxoSmithKlineCompletedWiskott-Aldrich SyndromeUnited States
-
GenethonHôpital Necker-Enfants MaladesCompleted