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Posted On: 03/20/2015 9:59:47 PM
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To date, the Foundation has funded research into Wolfram Syndrome, being led by Dr. Fumihiko Urano at Washington University Medical Center, which is now ready for clinical trials. The research team is pursuing several distinct types of treatments: The first is a pill, already approved by the FDA for other uses, which may help slow the progression of Wolfram Syndrome. It already has been studied in mouse models, with promising results, notes Dr. Saad Naseer, CEO/CMO of the Snow Foundation. “It’s really just a money issue. We’ve already identified the population for clinical trials...Within five years, we have a great potential for having something on the market. I believe that wholeheartedly.” He says about $5 to $7 million must be raised for the clinical trials to move forward. A second type of treatment in the works seeks to help patients regenerate cells that have been destroyed by the disease, which could actually reverse the effects of neurodegeneration, Urano adds—though that treatment will take longer to study and develop.
To date, the Foundation has funded research into Wolfram Syndrome, being led by Dr. Fumihiko Urano at Washington University Medical Center, which is now ready for clinical trials. The research team is pursuing several distinct types of treatments: The first is a pill, already approved by the FDA for other uses, which may help slow the progression of Wolfram Syndrome. It already has been studied in mouse models, with promising results, notes Dr. Saad Naseer, CEO/CMO of the Snow Foundation. “It’s really just a money issue. We’ve already identified the population for clinical trials...Within five years, we have a great potential for having something on the market. I believe that wholeheartedly.” He says about $5 to $7 million must be raised for the clinical trials to move forward. A second type of treatment in the works seeks to help patients regenerate cells that have been destroyed by the disease, which could actually reverse the effects of neurodegeneration, Urano adds—though that treatment will take longer to study and develop.
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