Raise the Volume for 5p- Syndrome

• Do you own a business which can offer its services?
• Do you have tickets to a Sporting or entertainment event?
• Do you have access to a vacation home or time-share rental?
• Are you a member of a local Golf Club?

We are reaching out to request any in-kind donation which we can utilize in our auction or raffles, which we believe would make a significant impact on our efforts. Your contribution would be an invaluable asset to our organization and would help us raise the necessary funds to continue our path forward for a treatment. In return, we would be happy to offer recognition at the event and on our social media platforms. We will also provide a receipt for the value of the donation for tax purposes.

We appreciate your consideration of our request, and welcome the opportunity to discuss this further with you. If you have any questions or need further information, please do not hesitate to contact us at 631-796-6070, or at Megan.Leston@criduchatresearch.org. Thank you for you generosity and support.

The Cri du Chat Research Foundation (CDCRF) is a 501(c)(3) nonprofit public charity committed to advancing translational research aimed at developing effective therapies for the significant neurological symptoms associated with 5p- syndrome, a rare and profoundly life-altering genetic disorder. Our mission is to enhance the quality of life, health, and independence of every child and adult affected by this condition—across the globe. 

What if a life-changing therapy—already FDA-approved—was sitting on a shelf today, waiting to be discovered, with the potential to dramatically improve the lives of individuals with 5p- syndrome?

To pursue that possibility, CDCRF has partnered with Charles River Laboratories, a global leader in rare disease research, to launch an ambitious drug repurposing screen. Together, we are testing more than 5,000 FDA-approved drugs and gene-targeting compounds using neurons derived from the cells of 5p- syndrome patients—the very cells most impacted by this disorder.

By focusing on therapies that already have known safety profiles, we aim to accelerate the path to clinical use, potentially delivering meaningful treatments to patients faster and more efficiently than traditional drug development pipelines.

This groundbreaking work also employs an innovative imaging technique known as cell painting, which generates a detailed biological "fingerprint" of 5p- syndrome from patient-derived cells. This fingerprint not only helps identify promising therapies, but also provides a powerful tool for measuring their impact with unprecedented precision.

Just as critically, the study will help to further identify and validate the key gene targets driving the disorder—an essential step toward developing truly disease-modifying therapies, including RNA and gene therapies, that could one day transform the future for individuals living with 5p- syndrome.

We have successfully funded and completed Stage One of this research. Now, we are working to raise the remaining $200,000 needed to complete Stage Two of this vital collaboration with Charles River Laboratories.

Join us in pushing the boundaries of what’s possible—and in making a profound, lasting difference in the lives of children living with 5p- syndrome.