Setbp1 Society

“We funded Unravel Biosciences with 38000 to generate primary RNA sequencing data to virtually screen over 40000 FDA approved and GRAS generally recognized as safe compounds The funded study aims to identify potential treatments mitigate adverse effets and accelerate the path to clinical trialsultimately expanding therapuetic possibilities for individuals with SETBP1-HD and SETBP1-RD Following this study identified therapeutics will be further evaluated in validated SETBP1 research models The University of Texas Southwestern Medical Center was funded 6250 to enable deeper understanding of how SETBP1 alters brain development cell identity and behavior via a preclinical mouse model of SETBP1 The University of Alabama at Birmingham was funded 6250 to identify drug repurposing candidates for SETBP1 using advanced computational approaches and publically avialable SETBP1-HD gene expression data The result of this study will be to provide another set of independently derived drug cancdidates for SETBP1-HD from already approved FDA drugs Hospital for Sick Children in Toronto Ontario Canada was funded 5643 USD to establish human neurons and brain organoid models of SETBP1 to reveal how the loss of gene impairs human neural cells and whether the symptoms are reversible Success of this trial could provide a foundation for future discoveries and advance the devlopment of future treatments”

“Other expenses include the following 1480 for insurance 1301 for awareness supplies to promote SETBP1 at a fundraising event hosted by the University of Pennsylvania for multiple rare diseases”