SYNGAP1-Related Disorder Therapeutic Pipeline

“When will there be a treatment for my child?” This is the first question asked by a newly diagnosed parent once they learn “What is SYNGAP1?” and find out there is no cure. Of course they ask, “When?” We all ask this question, and although we can’t pinpoint a date, we can share hope that treatments are on the way. SynGAP Research Fund invests in numerous possible approaches to find a cure, many of which are noted in the graphic below. The arrows show the stage each is at currently.

Although there is no date to which we can point as when we will have medicines to cure every Syngapian in the world, rest assured that this outcome is our goal. We can also take heart at the advances we have made since SYNGAP1-Related Disorder was first discovered to cause ID in 2009. Although 15 years seems too long to go without a cure, we continue to get closer every single year with the help of our partners, sponsors, scientists, clinicians, grantees, volunteers, and especially to everyone who participates in surveys, registries, and studies. All of these accelerate progress toward a cure. All of these are crucial pieces of our future.

If some of the terminology in the chart above doesn’t make sense, there is sure to be mention of most somewhere on our website. Rather than list everything here, you can use our site search engine to find information. Better yet, watch the video below of SRF Managing Director Mike Graglia clarify where we are at and what you can expect in the next year or two.

From SYNGAP1 Conference hosted by SRF, 2023 in Orlando

We are living in a very exciting time, finding more patients every quarter and getting closer and closer to treatments. Stay up to date and follow SRF everywhere for the latest updated information, and thank you all for your assistance in getting us where we are today!