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Hemofilie B; nog steeds on track?

317 Posts
Pagina: «« 1 ... 11 12 13 14 15 16 | Laatste | Omlaag ↓
  5. flosz 5 maart 2020 19:07

    • flosz

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    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

    haemophilia.ie/first-person-with-haem...

    www.irishtimes.com/news/ireland/irish...

    twitter.com/kev_omahony/status/123555...
  6. Vitavita 25 juni 2020 17:16

    • Vitavita

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    ir.catalystbiosciences.com/news-relea...

    "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."
  7. flosz 4 november 2020 16:25

    • flosz

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    $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 twitter.com/floszcrxl/status/13240075...

    $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
    twitter.com/floszcrxl/status/13240077...

    $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
    twitter.com/floszcrxl/status/13240079...
    Bijlage:
  14. flosz 1 september 2021 21:23

    • flosz

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    What to expect at the FDA's two-day meeting on gene therapy safety

    www.biopharmadive.com/news/fda-gene-t...

    Food and Drug Administration (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) Meeting #70
    Toxicity Risks of Adeno-associated Virus (AAV) Vectors for Gene Therapy (GT)
    September 2-3, 2021

    www.fda.gov/media/151599/download
  18. forum rang 10 DeZwarteRidder 29 februari 2024 17:17

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    Goldman Sachs slashes uniQure stock PT to $8 from $63.00 amid AMT-130 concerns
    Editor Ismeta Mujdragic Stock Markets

    Published Feb 29, 2024 09:06AM ET
317 Posts
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