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Hemofilie B; nog steeds on track?

317 Posts, Pagina: « 1 2 3 4 5 6 ... 11 12 13 14 15 16 | Laatste
flosz
0
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...
Vitavita
0
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."
flosz
0
$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:
flosz
0
Art. 29 juni, 2020. Freeline.

Dangerously high FIX levels reported in #hemophiliaB gene therapy.
A patient reported his FIX levels had risen to over 500% of normal after receiving AAV-S3 (FLT-180) $FRLN chscontact.ca/dangerously-high-fix-le...
Bijlage:
flosz
0
Gene therapies for the rare blood clotting disorder hemophilia B have taken a step closer to the market as Dutch developer $QURE became the first to release interim phase III results last week. www.labiotech.eu/gene-cell-therapy/un...

twitter.com/profmakris/status/1336593...
flosz
0
Ter aanvulling.....( tweet Prof. Makris).

Table adapted from Srivasta A, et al. Hemophilia. 2020;26:1-158
WFH Guidelines for the Management of Hemophilia, 3rd edition.
P.21. onlinelibrary.wiley.com/doi/epdf/10.1...

twitter.com/floszcrxl/status/13366773...
flosz
0
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
DeZwarteRidder
0
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, Pagina: « 1 2 3 4 5 6 ... 11 12 13 14 15 16 | Laatste
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