Click here for the ISTH 6 May 2025 Call to Action: Critical Juncture in Hemophilia Treatment. Thanks to Elaine Benoit, Precision BioLogic Inc. for the heads-up. Please post your comments below.
From Bob Gosselin, 7 May 2025: This link is more of a political statement than any notion of gene Rx acceptance treatment in HA or HB. This ISTH referendum is noting how only high-income countries can afford the existing gene therapy and therefore not available to all worldwide, so changes should be made to facilitate that Rx availability. This has nothing to do with “acceptance”. I was wondering whether this link was providing some guidance for post-gene Rx testing, as the gain-in-function of these therapies also show a discrepancy between testing platforms, whether it be OSA or chromo testing…at least that is what I saw for FIX gene Rx.
From Geo: thank you, Bob. I’ve seen pubs that also indicate standard OSA or CSA results don’t match predicted (calculated) results based on dosage and PK, presumably because the induced FVIII is produced by hepatocytes rather than Kuppfer cells.
No comments here.