Thank you for sharing this information to such a great audience. We think this could revolutionize the way we incentivize the development of new treatments, and reduce healthcare costs!
Thank you for all your work to find treatments for rare diseases. My daughter has Wolfram Syndrome, a rare form of diabetes that also causes vision loss, hearing loss, and other neurodegenerative problems. Currently, a repurposed drug has been identified that halts cell death during pre-clinical trials in animal and cell models, but there is no funding to proceed with clinical trials in Wolfram patients. The significance of this trial is that it could lead to a cure for all forms of diabetes regardless of causative factors. Just think how much money would be saved by governmental healthcare agencies if there were a cure for diabetes! Please consider Wolfram Syndrome as one of your projects for the pilot program in the UK. There is an active medical community studying Wolfram Syndrome in the UK and a strong advocacy group as well. For more detailed information on the Wolfram research, you may contact Dr. Saad Naseer, CMO, The Snow Foundation, .(JavaScript must be enabled to view this email address). I look forward to hearing from you.
Dear Gina-thanks for your comment. We actually have been looking at drug repurposing for Wolfram Syndrome, and have a couple of proposals that we are reviewing at the moment. I will contact Dr. Naseer to see if these include the one you described, or they are additional drug repurposing opportunities.
Great! It feels like we are so close with finding a therapeutic solution for Wolfram Syndrome. I am appreciative of your work and encouraged that many will benefit from it. Thank you again.
COMMENTS
BY Bruce Bloom
ON April 8, 2016 11:49 AM
Thank you for sharing this information to such a great audience. We think this could revolutionize the way we incentivize the development of new treatments, and reduce healthcare costs!
BY Gina Wipperman
ON April 9, 2016 08:31 AM
Thank you for all your work to find treatments for rare diseases. My daughter has Wolfram Syndrome, a rare form of diabetes that also causes vision loss, hearing loss, and other neurodegenerative problems. Currently, a repurposed drug has been identified that halts cell death during pre-clinical trials in animal and cell models, but there is no funding to proceed with clinical trials in Wolfram patients. The significance of this trial is that it could lead to a cure for all forms of diabetes regardless of causative factors. Just think how much money would be saved by governmental healthcare agencies if there were a cure for diabetes! Please consider Wolfram Syndrome as one of your projects for the pilot program in the UK. There is an active medical community studying Wolfram Syndrome in the UK and a strong advocacy group as well. For more detailed information on the Wolfram research, you may contact Dr. Saad Naseer, CMO, The Snow Foundation, .(JavaScript must be enabled to view this email address). I look forward to hearing from you.
BY Bruce Bloom
ON April 11, 2016 09:55 AM
Dear Gina-thanks for your comment. We actually have been looking at drug repurposing for Wolfram Syndrome, and have a couple of proposals that we are reviewing at the moment. I will contact Dr. Naseer to see if these include the one you described, or they are additional drug repurposing opportunities.
BY Gina Wipperman
ON April 11, 2016 01:34 PM
Great! It feels like we are so close with finding a therapeutic solution for Wolfram Syndrome. I am appreciative of your work and encouraged that many will benefit from it. Thank you again.