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What's the potential of Global Blood Therapeutics’ Voxelotor (GBT440) in sickle cell disease after data released at ASH 2017Ticker(s): GBT
Name: Dr John Strouse - MD
Institution: Duke University
- Associate Professor of Medicine and Pediatrics and Director of the Adult Sickle Cell Program at Duke University.
- Currently cares for 200 patients with Sickle Cell Disease (100 children & 100 adults) and15 patients with hereditary hemochromatosis.
- Research has focused on the epidemiology, risk factors, and prevention of the pulmonary and central nervous system complications of sickle cell disease and includes retrospective and prospective cohort studies and clinical trials; local site co-investigator on trials for GBT-440 and rivapansel.
Can you describe your experience treating patients with sickle cell disease? What is the first line of therapy that you use, and what are the pros and cons of each of those treatments?Added By: c_admin
Can you describe mechanism of action for GBT440? Are there any other hemoglobin oxygen affinity modulators out there, and how do they differ from GBT440? From a mechanistic perspective, how does GBT440 relieve sickle cell crises and pain?Added By: c_admin
The Phase1/2 study showed that 46% of patients in the treatment group demonstrated an increase in hemoglobin (greater than 1 g/dL increase). How meaningful are these results for you?Added By: c_admin
In a case study of a male age 67, reductions were observed in reticulocyte count and bilirubin, and blood oxygen levels were up from 86mmHg at baseline to 96mmHg at 65 weeks. Are those clinically significant results, and if so, what is the likelihood of this being replicated across all patients of all ages in the HOPE studies?
On a scale from 1-10, 10 being extremely excited, how would you rate your level of excitement for this drug?Added By: c_admin
What is the typical treatment regimen of your current patient population? Assuming Voxelotor is approved how likely will the prescribing community switch to Voxelotor or will there be a gradual shift as consensus builds for the products use?Added By: alexander poulos
How much of a competitive threat do you view Lentiglobin assuming bluebird bio is successful in bringing it to market?Added By: alexander poulos
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