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Preparing for upcoming 3 year data on BioMarin's gene therapy ValRox in treating patients with hemophilia ATicker(s): BMRN, ONCE, PFE
Institution: University of Southern California
- Director of the Hemostasis and Thrombosis Center at Children’s Hospital Los Angeles, Professor of Pediatrics at USC & Chair of the Scientific and Standardization Committee on Factor VIII, Factor IX, and Rare Bleeding Disorders of the International Society on Thrombosis and Haemostasis.
- Currently manages 320 patients with hemophilia A and B
- Research activities encompass clinical trials in hemophilia, the use of novel anticoagulants in children, and the development of the global hemostasis assays to monitor the effects of medications used to treat bleeding in hemophilia.
Please describe your clinical practice; roughly how many patients with hemophilia do you currently manage?Added By: joe_mccann
What are your expectations for the 3 years outcomes data to be presented at the upcoming ISTH meeting?Added By: joe_mccann
The median factor VIII activity level was 89% after 1 year, but drifted down to 46% after 2 years.
- What is the minimum medium level you need to see here to be enthusiastic about the program?
- Do you need to see a slowing rate of decline? By how much?
- Is there any chance it could improve again by year 3, or is there only a chance to get worse?
- If it stays about the minimum threshold to induce clotting is that enough?
If patients median factor VIII activity level continues to decline, how many years above the minimum threshold would be appealing to you? Your patients?Added By: joe_mccann
What other key endpoints, beyond median factor VIII activity level are you most focused on?Added By: joe_mccann
Do you view annual bleed rate or median Factor VIII activity levels to be a more important endpoint at the 3 year mark?Added By: joe_mccann
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