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Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy.
von Drygalski, Annette; Gomez, Esteban; Giermasz, Adam; Castaman, Giancarlo; Key, Nigel S; Lattimore, Susan U; Leebeek, Frank W G; Miesbach, Wolfgang A; Recht, Michael; Gut, Robert; Dolmetsch, Ricardo; Monahan, Paul E; Le Quellec, Sandra; Pipe, Steven W.
Afiliación
  • von Drygalski A; Division of Hematology/Oncology, Department of Medicine, University of California San Diego, San Diego, CA.
  • Gomez E; Center for Inherited Blood Disorders, Orange, CA.
  • Giermasz A; Division of Hematology/Oncology, Department of Medicine, Hemophilia Treatment Center, University of California Davis, Sacramento, CA.
  • Castaman G; Center for Bleeding Disorders, Department of Oncology, Careggi University Hospital, Florence, Italy.
  • Key NS; Division of Hematology and Blood Research Center, Department of Medicine, University of North Carolina, Chapel Hill, NC.
  • Lattimore SU; The Hemophilia Center, Oregon Health & Science University, Portland, OR.
  • Leebeek FWG; Department of Hematology, Erasmus MC, University Medical Center, Rotterdam, The Netherlands.
  • Miesbach WA; Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt, Germany.
  • Recht M; American Thrombosis and Hemostasis Network, Rochester, NY.
  • Gut R; Hemophilia Treatment Center, Yale University School of Medicine, New Haven, CT.
  • Dolmetsch R; uniQure Inc, Lexington, MA.
  • Monahan PE; uniQure Inc, Lexington, MA.
  • Le Quellec S; CSL Behring, King of Prussia, PA.
  • Pipe SW; CSL Behring Europe, Hattersheim am Main, Germany.
Blood Adv ; 7(19): 5671-5679, 2023 10 10.
Article en En | MEDLINE | ID: mdl-36490302

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Hemofilia B Tipo de estudio: Clinical_trials / Guideline / Prognostic_studies Límite: Adult / Humans Idioma: En Año: 2023 Tipo del documento: Article

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Hemofilia B Tipo de estudio: Clinical_trials / Guideline / Prognostic_studies Límite: Adult / Humans Idioma: En Año: 2023 Tipo del documento: Article