Looking for a Hit: 3000 donors for 3000 drugs
RSRT has committed to testing every FDA-approved drug for efficacy in treating symptoms of Rett Syndrome.
What is a large-scale drug screen, and why must it be undertaken?
Clinical trials are the endpoint of a drug discovery process that typically takes 12 years and costs many hundreds of millions of dollars. A possible way to shortcut this process is to identify existing drugs that have the ability to improve Rett symptoms.
The Trust has therefore approved the careful and methodical screening of all 2000 FDA-approved drugs plus 1000 compounds. “Hits” (drugs or compounds showing improvements) will be immediately evaluated and fast-tracked directly to clinical trials.
Project
The drug screen is being undertaken in the laboratory of Andrew Pieper, M.D., Ph.D. at University of Texas Southwestern Medical Center Dallas with the support of Steven McKnight, Ph.D. The Rett mice will be assessed for any improvement in a broad spectrum of symptoms.
This is crucial work that is ready to go. We call out to every member of the Rett community and beyond to speed this project quickly towards completion by sponsoring a drug. Our children deserve a better future. Help us take an essential step forward.
Click here to learn about our Family Challenge and add a name/photo.
Please consider one of these gifts:
$168 sponsors 1 drug
$336 sponsors 2 drugs
$504 sponsors 3 drugs
