Cri Du Chat Research Foundation

Cri Du Chat Research Foundation CDCRF is a 501c3 Non-Profit supporting the development of a treatment for Cri du Chat / 5p- Syndrome. It impacts 1 in 15,000 newborns annually.

In July 2014, The Cri du Chat Research Foundation (CDCRF) was founded by Megan and JC Leston, parents to their son Liam, who was diagnosed with Cri du Chat / 5p Minus syndrome in October 2013. Our mission was to accelerate the advancement of research on Cri du Chat / 5p Minus Syndrome with the commitment to developing a medical treatment, such as a Gene therapy (replacing or repairing the function

of a targeted gene or genes by introducing specific genetic material) or RNA therapy (a new class of medication, like the coronavirus vaccines) which would create an impact at the genetic level for the CdCS Individual to minimize or possibly cure symptoms of Cri du Chat Syndrome. Cri du Chat Syndrome is a severe rare neuro-developmental syndrome that is caused by a deletion of genes on the 5th chromosome. The Syndrome causes many challenges, including muscle weakness and coordination deficits, apraxia of speech, swallowing issues and autism. Some children have major organ involvement such as heart and kidney disease, or suffer from brain seizures. The diagnosis of CdCS / 5p Minus syndrome is a spectrum disorder due to the wide range of symptoms, but all individuals require lifelong support even with today's interventions and therapies. Over recent years, through our efforts, the Cri du Chat Research Foundation has achieved:
• Funding a Gene Therapy program using CRISPRa technology at the Ahituv Lab in University of California San Francisco
• Funding a Gene Therapy program using Stem Cell technology at University of California Davis
• Funding a RNA Therapy program using A*O technology at iXCells Biotechnologies
• Partnered with Simons Searchlight to collect data to utilize for a Natural History study of Cri du Chat Syndrome
(a requirement to proceed with Clinical trials)
• Collaborating with the Chung Lab at the Boston Children's Hospital to evaluate critical genes within CdCS/5p-

The Foundation's years of support for innovative research has created an opportunity for the scientific breakthrough needed for this rare genetic disease and real promise for changing the lives of those impacted by Cri du Chat syndrome.

The Cri du Chat Research Foundation and COMBINEDBrain - Outcome Measures and Biomarkers for Neurodevelopmental will be c...
06/04/2026

The Cri du Chat Research Foundation and COMBINEDBrain - Outcome Measures and Biomarkers for Neurodevelopmental will be collecting research specimens during the 5p- Society Conference in San Antonio.

We are currently seeking participation from individuals with genotype-only deletions and mosaic 5p- syndrome. Every sample collected helps strengthen the research foundation needed to better understand 5p- syndrome, identify biomarkers for the disorder, and accelerate the path toward disease-modifying therapies.

📅 Saturday, July 25
⏰ 7:30 AM – 12:30 PM
📍 Marriott Hotel, San Antonio

Every family that chooses to engage in research plays a valuable role in moving the field forward. We are profoundly grateful to the 5p- syndrome community for standing together in support of discovery, hope, and progress. Every contribution helps move us closer to a brighter, more equitable future for individuals and families living with 5p- syndrome

Today is International Cri du Chat Awareness Day, a special day to recognize, celebrate, and honor the incredible indivi...
05/05/2026

Today is International Cri du Chat Awareness Day, a special day to recognize, celebrate, and honor the incredible individuals and families in the Cri du Chat/5p- Syndrome community around the world.

Today, we celebrate the strength, resilience, joy, and determination of every person living with 5p- syndrome. We celebrate the parents, siblings, grandparents, and all caregivers who show up every day and work tirelessly to help individuals with 5p- syndrome live their best lives

Most of all, we celebrate a community that continues to inspire hope through love, perseverance, and unity.

At the Cri du Chat Research Foundation, our mission is rooted in that hope. We are committed to advancing research, deepening the understanding of 5p- syndrome, and helping accelerate the development of meaningful treatments that can improve lives.

We also believe this community deserves equity in scientific progress and access to the same innovation, urgency, and treatment development seen across other conditions. Families deserve answers, progress, and the promise of a brighter future.

In honor of International Cri du Chat Awareness Day, we are also proud to host our Annual Charity Golf Outing on May 12 to raise critical funds for research. This special event helps fuel the science, advocacy, and momentum needed to create real change for this community.

We invite you to support by becoming a sponsor or making a direct donation. Learn more here: https://secure.qgiv.com/for/cdcrfgolfouting/event/golf2026/

International Cri du Chat Awareness Day is also a reminder that rare does not mean alone. To every family on this journey: we see you, we support you, and we stand with you.

Happy International 5p- Syndrome Awareness Day!

This full-day charity golf tournament supports the Cri Du Chat Research Foundation with a complete schedule of activities from 9:30 AM to 6:30 PM. The event features morning check-in and brunch, followed by a golf scramble tournament with shotgun start, and concludes with "The Giving Green Ceremony"...

04/14/2026

Natural history studies are an important way to help researchers better understand 5p- syndrome and support future research.

The Cri-du-Chat Research Foundation collaborates with two studies that families can participate in from home: Citizen Health and Simons Searchlight. Both platforms prioritize privacy and data security, with de-identified data and protections in place for participants.

Learn more and sign up using the links in our bio.

04/01/2026
Stay connected and never miss an update!Sign up for the Cri du Chat Research Foundation newsletter to get the latest new...
03/31/2026

Stay connected and never miss an update!

Sign up for the Cri du Chat Research Foundation newsletter to get the latest news, research updates, and ways to get involved.

Join here: https://forms.gle/GVhp6rCGrBYe5TZp7

Thank you to everyone who has registered and participated in Simons Searchlight.If you’ve already registered, don’t forg...
03/31/2026

Thank you to everyone who has registered and participated in Simons Searchlight.
If you’ve already registered, don’t forget to log back in and upload lab reports & complete your surveys. This is where your impact really counts!
If you haven’t signed up yet, we encourage you to sign up through the link in our bio.

A Disease Concept Model (DCM) study helps researchers better understand a condition by identifying the symptoms, challen...
03/26/2026

A Disease Concept Model (DCM) study helps researchers better understand a condition by identifying the symptoms, challenges, and impacts that matter most to individuals living with the condition—and to their caregivers. These insights are essential for improving treatments and ensuring clinical trials measure outcomes that will truly make a difference.

The Cri du Chat Research Foundation is currently recruiting caregivers of individuals with Cri du chat syndrome (deletion only genotype) to participate in a ~90-minute interview conducted by a genetic counseling master's student. This study aims to deepen our understanding of what matters most when treating symptoms and evaluating meaningful change in clinical trials.

*Other genotypes will take place in future Disease Concept Model studies- stay tuned for further information*

🗓 Interviews will take place during the summer of 2026.

If you’re interested in participating, please contact: [email protected]

Your voice can help shape the future of care and research 💙
COMBINEDBrain - Outcome Measures and Biomarkers for Neurodevelopmental

Attention 5p- Syndrome Families— COMBINEDBrain - Outcome Measures and Biomarkers for NeurodevelopmentalCheck out the Com...
03/20/2026

Attention 5p- Syndrome Families—
COMBINEDBrain - Outcome Measures and Biomarkers for Neurodevelopmental

Check out the CombinedBrain 2026 Roadshow Schedule!

If you’re located near one of the participating sites and are interested in contributing to research, we invite you to get involved. Families can help by providing specimens (such as blood or urine) to support ongoing studies.

To learn more or arrange free participation for your loved one with 5p- syndrome, please contact [email protected].

Your involvement can help drive critical research forward.

Inspiring conversations are taking place today in NYC at the CNBC Cures meeting on rare diseases. Visionary leaders from...
03/03/2026

Inspiring conversations are taking place today in NYC at the CNBC Cures meeting on rare diseases. Visionary leaders from across healthcare have come together with a shared determination to make this century a defining era of medical breakthroughs.

Thank you to Becky Quick and CNBC Cures for elevating this critical dialogue and uniting the pioneers who are shaping the future of medicine.

We’re grateful for your leadership in convening such an important gathering.


Watch live here…

CNBC Cures is a purpose-led initiative, with proceeds from sponsorships, event ticket sales, and related revenue reinvested back into CNBC Cures editorial operations.

02/28/2026

Rare Disease Day 2026
Cri du Chat Research Foundation

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