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Kliniske forsøg med Kreatinmangel, X-bundet
I alt 1561 resultater
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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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Eunice Kennedy Shriver National Institute of Child...AfsluttetFragilt X syndrom | For tidlig ovariesvigt | For tidlig overgangsalder | FMR1 præmutation | Primær Ovairan InsufficiensForenede 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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REGENXBIO Inc.Aktiv, ikke rekrutterendeMucopolysaccharidosis Type II (MPS II)Forenede Stater, Brasilien
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ShireAfsluttetHunters syndromForenede Stater, Det Forenede Kongerige
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ShireAfsluttetHunters syndrom | Mucopolysaccharidosis (MPS)Forenede Stater, Argentina, Mexico, Spanien, Det Forenede Kongerige
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National Institute of Neurological Disorders and...AfsluttetMotor neuron sygdomForenede Stater
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Psyadon PharmaAfsluttetLesch-Nyhans sygdomForenede Stater
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TakedaTakeda Development Center Americas, Inc.RekrutteringHunters syndrom | Mucopolysaccharidosis (MPS)Forenede Stater
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National Institute of Neurological Disorders and...RekrutteringMotor neuron sygdom | Spinal og Bulbar muskelatrofi | Kennedys sygdomForenede Stater
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ShireAfsluttetHunters syndromDet Forenede Kongerige, Forenede Stater, Brasilien
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Great Ormond Street Hospital for Children NHS Foundation...RekrutteringAlvorlig kombineret immundefekt, X-forbundetDet Forenede Kongerige
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REGENXBIO Inc.Aktiv, ikke rekrutterendeMucopolysaccharidosis Type II (MPS II)Forenede Stater, Canada
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Lundquist Institute for Biomedical Innovation at...AfsluttetMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VIForenede Stater
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Children's Hospital of Chongqing Medical UniversityRekruttering
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Cyprium Therapeutics, Inc.Tilmelding efter invitation
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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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Emalex Biosciences Inc.Psyadon PharmaAfsluttetLesch-Nyhans sygdom | Selvskadende adfærdSpanien, Forenede Stater
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Baylor College of MedicineUniversity of California, San Diego; University of Alabama at Birmingham; Boston Children's Hospital og andre samarbejdspartnereAfsluttetPrader-Willi syndrom | Rett syndrom | Angelman syndrom | SøvnproblemerForenede Stater
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Eunice Kennedy Shriver National Institute of Child...Afsluttet
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ShireCovance; PRA Health Sciences; PharmaNetAfsluttetMucopolysaccharidosis II | Hunters syndrom | MPS IIBrasilien, Polen, Taiwan
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University of California, San DiegoBioMarin PharmaceuticalTrukket tilbageAdfærdsmanifestationer af Lesch-Nyhans sygdomForenede Stater
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TakedaRekrutteringHunters syndromCanada, Frankrig
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CENTOGENE GmbH RostockAfsluttetMucopolysaccharidosis II | Hunters syndrom | Jægers syndrom, mild form | Hunter's Canal SyndromeMexico
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National Institute of Neurological Disorders and...AfsluttetMotor neuron sygdom | Spinal og bulbar muskelatrofi (SBMA)Forenede Stater
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National MPS SocietyMediResource Inc.AfsluttetMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidoser | Mucopolysaccharidosis VI | Mucopolysaccharidosis IVForenede Stater
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Manchester University NHS Foundation TrustAfsluttetMucopolysaccharidosis Type IIDet Forenede Kongerige
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Kunming Hope of Health HospitalAfsluttet
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Lundquist Institute for Biomedical Innovation at...AfsluttetMucopolysaccharidosis type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VIForenede Stater
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The Chaim Sheba Medical CenterRekrutteringFragilt X syndrom | Williams syndrom | Velokardiofacialt syndromIsrael
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The Hospital for Sick ChildrenAfsluttetX-bundet lymfoproliferativt syndrom type 2 (XLP-2)Canada
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Minoryx Therapeutics, S.L.Aktiv, ikke rekrutterendeAdrenoleukodystrofiSpanien, Forenede Stater, Ungarn, Frankrig, Tyskland, Italien, Holland, Det Forenede Kongerige
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National Taiwan University HospitalAfsluttet
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Masonic Cancer Center, University of MinnesotaTrukket tilbage
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Assistance Publique - Hôpitaux de ParisCentre National de la Recherche Scientifique, FranceAfsluttetAmyotrofisk lateral sklerose | Primær lateral sklerose | Kennedys sygdomFrankrig
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University Hospital HeidelbergAfsluttetMucopolysaccharidosis type I | Mucopolysaccharidosis Type II | Mestringsadfærd | Mucopolysaccharidosis Type III | AdfærdsforstyrrelserTyskland
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FDA Office of Orphan Products DevelopmentHugo W. Moser Research Institute at Kennedy Krieger, Inc.Afsluttet
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Far Eastern Memorial HospitalNational Taiwan University Hospital; Fu Jen Catholic UniversityUkendtAdrenoleukodystrofiTaiwan
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National Center for Research Resources (NCRR)Ohio State UniversityAfsluttetAmyotrofisk lateral sklerose | Spinal muskelatrofi | Spinobulbar muskelatrofi
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Institute for Neurodegenerative DisordersAfsluttetParkinsons sygdom | Alzheimers sygdom | Mild kognitiv svækkelse | Fragilt X syndrom | Huntingtons sygdom | Autistiske Spektrum ForstyrrelserForenede Stater
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ShireAfsluttetHunters syndrom | Mucopolysaccharidosis II (MPS II)Forenede Stater, Frankrig, Det Forenede Kongerige, Tyskland, Sverige, Spanien, Brasilien, Canada, Italien, Rumænien
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Hugo W. Moser Research Institute at Kennedy Krieger...AfsluttetAdrenoleukodystrofiForenede Stater
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MedDay Pharmaceuticals SAAfsluttetAdrenoleukodystrofi | Adrenomyeloneuropati | AMNFrankrig, Tyskland, Spanien
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Eunice Kennedy Shriver National Institute of Child...Food and Drug Administration (FDA)AfsluttetAdrenoleukodystrofi | AdrenomyeloneuropatiForenede Stater
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Masonic Cancer Center, University of MinnesotaRekruttering
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Minoryx Therapeutics, S.L.RekrutteringCerebral adrenoleukodystrofi (cALD)Forenede Stater, Brasilien, Argentina
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Poxel SAIkke rekrutterer endnu