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Kliniske forsøg med X-bundet myotubulær myopati
I alt 1536 resultater
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Sangamo TherapeuticsAktiv, ikke rekrutterendeHæmofili B | Mucopolysaccharidosis I | Mucopolysaccharidosis IIForenede Stater
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Massachusetts General HospitalNorthwestern University; University of Florida; University of Minnesota; Henry... og andre samarbejdspartnereRekrutteringALS | Kennedys sygdom | MND (motorisk neuronsygdom) | PLS | PMA - Progressiv muskelatrofi | PBP - Progressiv Bulbar PareseForenede Stater, Italien
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) og andre samarbejdspartnereAfsluttetMucopolysaccharidosis type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VI | Mucopolysaccharidosis Type IV | Mucopolysaccharidosis Type VIIForenede Stater, Canada
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Massachusetts General HospitalEuropean Leukodystrophy AssociationRekrutteringRestless Legs Syndrome | AdrenoleukodystrofiForenede Stater, Holland
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Massachusetts General HospitalNational Institute of Arthritis and Musculoskeletal and Skin Diseases...RekrutteringX-bundet hypofosfatæmi | Hypofosfatæmisk rakitt | Hypophosphatemisk rakitt, X-Linked DominantForenede Stater
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Minoryx Therapeutics, S.L.Aktiv, ikke rekrutterendeCerebral adrenoleukodystrofiSpanien, Tyskland, Frankrig, Argentina
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Great Ormond Street Hospital for Children NHS Foundation...National Institute for Health Research, United Kingdom; The Freya FoundationRekrutteringPyruvat dehydrogenase kompleks mangel | Pyruvat dehydrogenase E1 alfa-mangel | Pyruvat Dehydrogenase E1-Beta mangel | Pyruvat dehydrogenase E2 mangel | Pyruvat Dehydrogenase Phosphatase mangelDet Forenede Kongerige
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University of MiamiJackson Health SystemIkke længere tilgængelig
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Retina Foundation of the SouthwestDSM Nutritional Products, Inc.; Foundation Fighting BlindnessAfsluttetRetinitis Pigmentosa | X-bundne genetiske sygdommeForenede Stater
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Great Ormond Street Hospital for Children NHS Foundation...UkendtX-bundet svær kombineret immundefektDet Forenede Kongerige
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Shenzhen Second People's HospitalGuangzhou Women and Children's Medical Center; Shenzhen UniversityRekrutteringAdrenoleukodystrofi | Metakromatisk leukodystrofiKina
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Gillette Children's Specialty HealthcareAfsluttetCerebral Parese | Muskeldystrofi | Osteopeni | Rett syndrom | Spinal muskelatrofi | Spina BifidaForenede Stater
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National Institute of Allergy and Infectious Diseases...RekrutteringX-bundet svær kombineret immundefekt (XSCID)Forenede Stater
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St. Jude Children's Research HospitalNational Heart, Lung, and Blood Institute (NHLBI); Assisi Foundation; California...SuspenderetSvær kombineret immundefektsygdom, X-bundetForenede Stater
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Assistance Publique - Hôpitaux de ParisAfsluttetX-bundet svær kombineret immundefektFrankrig
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Murdoch Childrens Research InstituteUniversity of Sydney; Sydney Children's Hospitals Network; Women's and Children... og andre samarbejdspartnereAktiv, ikke rekrutterendeAutosomal recessiv lidelse | X-forbundne genetiske sygdommeAustralien
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Children's Hospital of PhiladelphiaRekrutteringLeukæmi | Knoglemarvssvigt syndromer | Immundefekter | Medfødte metabolismefejl | Immunodysregulering Polyendokrinopati Enteropati X-linked SyndromeForenede Stater
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National Institute of Allergy and Infectious Diseases...AfsluttetVækstsvigt | X-bundet svær kombineret immundefekt (XSCID) | VæksthormonresistensForenede Stater
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Edimer PharmaceuticalsAfsluttetX-bundet hypohidrotisk ektodermal dysplasiForenede Stater
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Masonic Cancer Center, University of MinnesotaAfsluttetSeglcellesygdom | Thalassæmi | Cerebral adrenoleukodystrofi | Arvelige stofskifteforstyrrelser | Højrisiko hæmatologiske lidelserForenede Stater
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University Hospital ErlangenEdimer PharmaceuticalsAfsluttetX-bundet hypohidrotisk ektodermal dysplasiTyskland
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Mitsubishi Tanabe Pharma America Inc.RekrutteringErytropoietisk protoporfyri (EPP) | X-forbundet protoporfyri (XLP)Forenede Stater, Spanien, Frankrig, Det Forenede Kongerige, Japan, Italien, Australien, Bulgarien, Tjekkiet, Holland, Polen
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Edimer PharmaceuticalsAfsluttetX-forbundet hypohidrotisk ektodermal dysplasiForenede Stater
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Hugo W. Moser Research Institute at Kennedy Krieger...RekrutteringNeurodegenerative sygdomme | Ataksi | Cerebellar ataksi | Leukodystrofi | Adrenomyeloneuropati | LBSL | Leukoencefalopati med hjernestamme og rygmarvsinvolvering og laktatforhøjelse | Leukoencefalopati med hjernestamme og rygmarvsinvolvering og højt laktatsyndrom (lidelse)Forenede Stater
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National Institute of Allergy and Infectious Diseases...AfsluttetGenetiske sygdomme, X-forbundet | Lymfoproliferativ sygdom | X-forbundet lymfoproliferativ sygdomForenede Stater
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Shanghai Children's HospitalRekrutteringAlport syndrom, X-LinkedKina
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David WilliamsAktiv, ikke rekrutterendeGenterapi | Alvorlig kombineret immundefekt, X-forbundetForenede Stater, Det Forenede Kongerige
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National Institute of Allergy and Infectious Diseases...UkendtX-forbundne kombinerede immundefektsygdommeForenede Stater
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Edimer PharmaceuticalsAktiv, ikke rekrutterendeX-bundet hypohidrotisk ektodermal dysplasiForenede Stater, Det Forenede Kongerige, Frankrig, Tyskland, Italien
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National Institute of Allergy and Infectious Diseases...AfsluttetAlvorlig kombineret immundefektForenede Stater
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Assistance Publique - Hôpitaux de ParisRekrutteringAdrenoleukodystrofi | Adrenomyeloneuropati | Metakromatisk leukodystrofi | Voksen-debut leukoencefalopati med axonale sfæroider og pigmenteret gliaFrankrig
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Great Ormond Street Hospital for Children NHS Foundation...RekrutteringAlvorlig kombineret immundefekt, X-forbundetDet Forenede Kongerige
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Children's Hospital of Chongqing Medical UniversityRekruttering
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Institut National de la Santé Et de la Recherche...RekrutteringMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Mucopolysaccharidosis III | Mucopolysaccharidosis IV | Mucopolysaccharidosis VII | Multipel Sulfatase mangel sygdom | Mucopolysaccharidosis IXFrankrig
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The Hospital for Sick ChildrenAfsluttetX-bundet lymfoproliferativt syndrom type 2 (XLP-2)Canada
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Stephen G. Kaler, MDRekrutteringMenkes sygdom | Occipital Horn SyndromForenede Stater
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Zhongmou TherapeuticsRekrutteringX-bundet retinoschisisKina
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Shanghai General Hospital, Shanghai Jiao Tong University...Innostellar BiotherapeuticsRekrutteringX-bundet retinoschisisKina
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Edimer PharmaceuticalsAfsluttetX-bundet hypohidrotisk ektodermal dysplasi | Hypohidrotisk ektodermal dysplasiForenede Stater
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Edimer PharmaceuticalsAfsluttetX-bundet hypohidrotisk ektodermal dysplasi | Hypohidrotisk ektodermal dysplasiSpanien
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Applied Genetic Technologies CorpAfsluttetX-bundet retinoschisisForenede Stater
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Atsena Therapeutics Inc.RekrutteringX-bundet retinoschisisForenede Stater
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Jakub AntczakAfsluttetAdrenomyeloneuropati | Arvelig spastisk paraplegiPolen
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IRCCS San RaffaeleFondazione TelethonRekrutteringKronisk granulomatøs sygdom X-bundet (X-CGD)Italien
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University of California, Los AngelesNational Heart, Lung, and Blood Institute (NHLBI); Boston Children's Hospital og andre samarbejdspartnereRekrutteringGranulomatøs sygdom, kronisk, X-bundetForenede Stater
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Applied Genetic Technologies CorpFoundation Fighting BlindnessAfsluttetX-bundet retinoschisis | XLRSForenede Stater
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University of California, San FranciscoRekrutteringMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Wolmans sygdom | Pompes sygdom infantil | Mucopolysaccharidosis IV A | Mucopolysaccharidosis VII | Neuronopatisk Gauchers sygdomForenede Stater
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University of CincinnatiChildren's Hospital Medical Center, CincinnatiAfsluttetAdrenoleukodystrofi | Zellwegers syndrom | Infantil Refsums Sygdom | Bifunktionel enzymmangel
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Bio Products LaboratoryAfsluttetAlmindelig variabel immundefekt | Primære immundefektsygdomme | Hyper-IgM syndrom | X-bundet agammaglobulinæmiForenede Stater, Det Forenede Kongerige, Ungarn
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Edimer PharmaceuticalsAfsluttetX-forbundet hypohidrotisk ektodermal dysplasiForenede Stater