The foundation is proud to announce after countless hours of research, meetings, and vetting proposals the start to the drug repurposing project with Rare Base. There is still a long road ahead of us with much work to be done and funds to be raised but we are starting on the path to a cure.
The foundation had consulted with other Rare Disease groups and in working with our independent scientific consultant made the decision to move forward with the Rare Base Proposal. They have been flexible and open from the start which helps invoke confidence in their methodology and willingness to help members in the rare disease community.
-Rare Base has vetted potential drug candidates to ensure that everything screened is approved for children, crosses the blood brain barrier (which we need), has a tangible percentage increase, and of course above all is safe
- Rare Base uses state of the art iPSCs that are engineered using CRISPR technology to express a disease-associated mutation.
-Rare base is willing and open to outside collaboration. Our independent scientific consultant has vetted possible options to help study and better understand the results to only further the research and advance the understanding of the YWHAG gene.
-The University of Alabama who sponsored the YWHAG study on the foundation’s website recommended Rare Base even though they have no formal relationship.
Image courtesy of Rare Base
While there are many unknowns that are ahead, we are on the journey to a cure and performing cutting edge research that will help all our families now and in the future.
Now is not the time to take our foot off the gas but to only press harder as we race towards a cure. We ask for help and support of our community to raise awareness and advocate for our ongoing fundraising efforts