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Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B.
Von Drygalski, Annette; Giermasz, Adam; Castaman, Giancarlo; Key, Nigel S; Lattimore, Susan; Leebeek, Frank W G; Miesbach, Wolfgang; Recht, Michael; Long, Alison; Gut, Robert; Sawyer, Eileen K; Pipe, Steven W.
Affiliation
  • Von Drygalski A; Division of Hematology/Oncology, Department of Medicine, University of California San Diego, San Diego, CA.
  • Giermasz A; Division of Hematology/Oncology, Department of Medicine, Hemophilia Treatment Center, University of California Davis, Sacramento, CA.
  • Castaman G; Careggi University Hospital, Florence, Italy.
  • Key NS; Division of Hematology/Oncology, Department of Medicine, University of North Carolina, Chapel Hill, NC.
  • Lattimore S; The Hemophilia Center, Oregon Health & Science University, Portland, OR.
  • Leebeek FWG; Erasmus University Medical Center, Rotterdam, The Netherlands.
  • Miesbach W; Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt, Germany.
  • Recht M; The Hemophilia Center, Oregon Health & Science University, Portland, OR.
  • Long A; uniQure Inc, Lexington, MA; and.
  • Gut R; uniQure Inc, Lexington, MA; and.
  • Sawyer EK; uniQure Inc, Lexington, MA; and.
  • Pipe SW; Department of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI.
Blood Adv ; 3(21): 3241-3247, 2019 11 12.
Article in En | MEDLINE | ID: mdl-31698454
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Blood Adv Year: 2019 Type: Article Affiliation country: Canada
Fulltext
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PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Blood Adv Year: 2019 Type: Article Affiliation country: Canada