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Hemofilie B; nog steeds on track?

307 Posts
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  1. flosz 5 maart 2020 19:07
    Etranacogene Dezaparvovec

    There’s been a medical breakthrough in the treatment of haemophilia

    The first person in Ireland with the condition has been treated using gene therapy
  2. Vitavita 25 juni 2020 17:16

    "Studies of CB 2679d-GT in hemophilia B mice have demonstrated a 4-fold reduction in blood loss and an 8-fold reduction in bleeding time when compared with the same dose of the Padua variant of FIX. Furthermore, when packaged in a proprietary chimeric AAV capsid, CB 2679d-GT demonstrated a clear dose response of high stable FIX levels across the three dose levels in hemophilia B mice."
  3. flosz 4 november 2020 16:25
    $QURE #ASH2020 A Single Administration of AAV5-hFIX in Newborn, Juvenile and Adult Mice Leads to Stable hFIX Expression up to 18 Months after Dosing

    $QURE #ASH2020 Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial

    $QURE #ASH2020 AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years
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