From the archives 2014: ... If we get positive
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Posted On: 02/09/2020 10:31:46 PM
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From the archives 2014:
... If we get positive preclinical data, we have a chance to get an orphan drug designation for MANF for Wolfram syndrome. ...
https://wolframsyndrome.dom.wustl.edu/orphan-...signation/
Fast forward Summer 2019:
today announced that positive results from in vitro and animal studies on MANF in the treatment of Wolfram Syndrome were presented at the 79th Annual Scientific Sessions of the American Diabetes Association.
The abstract entitled ‘Mesencephalic Astrocyte-Derived Neurotrophic Factor (MANF): A New Therapeutic Target for Wolfram Syndrome,’ describes positive results that show MANF:
1. Activates proliferation of beta cell in WFS1 genetic animal models of Wolfram Syndrome
2. Suppresses key apoptosis and proteins misfolding
3. Activates the pro-survival and pro-protein folding mTOR/S6K signaling pathway
“MANF appears to have significant potential as a therapeutic agent for Wolfram Syndrome,” said Stephanie Gebel, Co-Founder and Chairman of the Board at the Snow Foundation, a co-sponsor of the research. “Going forward, we will be working with Amarantus to help bring this exciting research into clinical development to help the pediatric patients who are affected by Wolfram Syndrome.”
https://www.biospace.com/article/amarantus-an...sociation/
... If we get positive preclinical data, we have a chance to get an orphan drug designation for MANF for Wolfram syndrome. ...
https://wolframsyndrome.dom.wustl.edu/orphan-...signation/
Fast forward Summer 2019:
today announced that positive results from in vitro and animal studies on MANF in the treatment of Wolfram Syndrome were presented at the 79th Annual Scientific Sessions of the American Diabetes Association.
The abstract entitled ‘Mesencephalic Astrocyte-Derived Neurotrophic Factor (MANF): A New Therapeutic Target for Wolfram Syndrome,’ describes positive results that show MANF:
1. Activates proliferation of beta cell in WFS1 genetic animal models of Wolfram Syndrome
2. Suppresses key apoptosis and proteins misfolding
3. Activates the pro-survival and pro-protein folding mTOR/S6K signaling pathway
“MANF appears to have significant potential as a therapeutic agent for Wolfram Syndrome,” said Stephanie Gebel, Co-Founder and Chairman of the Board at the Snow Foundation, a co-sponsor of the research. “Going forward, we will be working with Amarantus to help bring this exciting research into clinical development to help the pediatric patients who are affected by Wolfram Syndrome.”
https://www.biospace.com/article/amarantus-an...sociation/
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