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Kliniske forsøg med X-Linked Charcot-Marie-Tooth Disease
I alt 1638 resultater
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS); Rare Diseases...RekrutteringPyruvat Dehydrogenase Complex Deficiency DiseaseForenede Stater
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Children's Hospital of PhiladelphiaEli Lilly and Company; University of Pennsylvania; Takeda; National Institute... og andre samarbejdspartnereRekrutteringMucopolysaccharidoser | Leukoencefalopati | Leukodystrofi | Adrenoleukodystrofi | Adrenomyeloneuropati | X-bundet adrenoleukodystrofi | Gangliosidoser | Metakromatisk leukodystrofi | Krabbes sygdom | Refsum sygdom | Cadasil | Sjögren-Larsson syndrom | Allan-Herndon-Dudley syndrom | Hvidstof sygdom | GM2 Gangliosidosis | Zellwegers... og andre forholdForenede Stater
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Children's Hospital of PhiladelphiaIllumina, Inc.Aktiv, ikke rekrutterendeMucopolysaccharidoser | Leukodystrofi | Adrenoleukodystrofi | Adrenomyeloneuropati | X-bundet adrenoleukodystrofi | Gangliosidoser | Metakromatisk leukodystrofi | Krabbes sygdom | Refsum sygdom | Cadasil | Sjögren-Larsson syndrom | Allan-Herndon-Dudley syndrom | Hvidstof sygdom | GM2 Gangliosidosis | Zellwegers syndrom | ALSP | Pelizaeus-Merzbachers sygdom og andre forholdForenede Stater
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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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National Human Genome Research Institute (NHGRI)RekrutteringMetabolisk sygdom | Purin-pyrimidin metabolisme | AICDA, OMIM *605257, Immundefekt med Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A, OMIM *606224, Anæmi, hæmolytisk, på grund af UMPH1-mangel | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064, Millers syndrom... og andre forholdForenede Stater
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VegaVect, Inc.National Eye Institute (NEI)Aktiv, ikke rekrutterendeRetinoschisis | X-LinkedForenede Stater
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National Eye Institute (NEI)AfsluttetRetinoschisis | X-LinkedForenede Stater
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West China HospitalRekruttering
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University of MiamiJackson Health SystemIkke længere tilgængelig
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Newcastle-upon-Tyne Hospitals NHS TrustRekrutteringX-bundet myotubulær myopati | Centronukleær myopati | Myotubulær myopati | Myotubulær myopati 1 | Myotubulær (Centronuclear) myopati | Centronuclear Myopati, X-LinkedDet Forenede Kongerige
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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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Astellas Gene TherapiesAfsluttetX-bundet myotubulær myopatiForenede Stater, Frankrig, Det Forenede Kongerige, Canada, Tyskland
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University of FloridaAudentes TherapeuticsAfsluttetX-bundet myotubulær myopatiForenede 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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Great Ormond Street Hospital for Children NHS Foundation...UkendtX-bundet svær kombineret immundefektDet Forenede Kongerige
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James DowlingCanadian Institutes of Health Research (CIHR); Cures Within Reach; The Joshua... og andre samarbejdspartnereRekrutteringX Linked Myotubular MyopatiDet Forenede Kongerige, Canada, Forenede Stater
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National Institute of Allergy and Infectious Diseases...RekrutteringX-bundet svær kombineret immundefekt (XSCID)Forenede Stater
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Assistance Publique - Hôpitaux de ParisAfsluttetX-bundet svær kombineret immundefektFrankrig
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Shenzhen Geno-Immune Medical InstituteUkendt
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Astellas Gene TherapiesAfsluttetMænd med X-bundet myotubulær myopati (XLMTM)Forenede Stater, Det Forenede Kongerige
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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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National Eye Institute (NEI)AfsluttetRetinoschisis | X-LinkedForenede 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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National Institute of Allergy and Infectious Diseases...AfsluttetGenetiske sygdomme, X-forbundet | Lymfoproliferativ sygdom | X-forbundet lymfoproliferativ sygdomForenede Stater
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University of California, DavisUniversity of Alberta; St. Justine's HospitalAfsluttetNeuroadfærdsmæssige manifestationer | Genetiske sygdomme, X-forbundet | Intellektuel handicap | Fragilt X syndrom | Kønskromosomforstyrrelser | Fragilt X Mental Retardation Syndrome | Trinukleotid Gentag Udvidelse | Fra(X) syndrom | FXS | Mental retardering, X LinkedForenede Stater, Canada
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Edimer PharmaceuticalsAfsluttetX-bundet hypohidrotisk ektodermal dysplasiForenede Stater
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Hospices Civils de LyonUkendtX-bundet mental retarderingFrankrig
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Cure CMDUniversity of Michigan; Congenital Muscle Disease International RegistrAfsluttet
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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, Japan, Det Forenede Kongerige, Italien, Australien, Polen, Bulgarien, Tjekkiet, Holland
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Edimer PharmaceuticalsAfsluttetX-forbundet hypohidrotisk ektodermal dysplasiForenede Stater
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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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National Institute of Allergy and Infectious Diseases...AfsluttetAlvorlig kombineret immundefektForenede Stater
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EspeRare FoundationIqvia Pty Ltd; Pierre Fabre MedicamentRekrutteringX-Linked Hypohidrotisk ektodermal dysplasi (XLHED)Forenede Stater, Spanien, Frankrig, Tyskland, Italien, Det Forenede Kongerige
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Edimer PharmaceuticalsAktiv, ikke rekrutterendeX-bundet hypohidrotisk ektodermal dysplasiForenede Stater, Det Forenede Kongerige, Frankrig, Tyskland, Italien
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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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The Hospital for Sick ChildrenAfsluttetX-bundet lymfoproliferativt syndrom type 2 (XLP-2)Canada
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IRCCS San RaffaeleFondazione TelethonRekrutteringKronisk granulomatøs sygdom X-bundet (X-CGD)Italien
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Icahn School of Medicine at Mount SinaiRekrutteringAutismespektrumforstyrrelse | DDX3X | Mental retardering, X-linked 102Forenede Stater
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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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Zhongmou TherapeuticsRekrutteringX-bundet retinoschisisKina
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Shanghai General Hospital, Shanghai Jiao Tong University...Innostellar BiotherapeuticsRekrutteringX-bundet retinoschisisKina
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Ovid Therapeutics Inc.AfsluttetFragilt X-syndrom (FXS)Forenede Stater
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Guido A. Davidzon, MD, SMTrukket tilbage
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Tetra Discovery PartnersAfsluttetFragilt X syndrom | Fra(X) syndrom | FXSForenede Stater
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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