CFC International

10/28/2026

As a member of Rasopathy Network, we are excited to share that Mount Sinai has announced BabyMERIT, the first NIH-funded U.S. phase 3 clinical trial to study the MEK inhibitor (Trametinib) in babies with life threatening CFC syndrome and other RASopathy-associated hypertrophic cardiomyopathy. This is a major milestone to celebrate.

We thank Dr. Bruce Gelb and Dr. Jane Newburger for their dedication and perseverance to make this study happen!

Moving beyond case studies, this prospective clinical trial is expected to generate rigorous evidence on the effects of using RAS pathway targeted drug treatments for a non-cancer indication in RASopathies. Proof of this concept will de-risk MEK inhibitor treatment and could even open doors to a broader scope of use.
RASopathies Network

09/23/2026

💙 UNSTOPPABLY BOLD DAY ROLL CALL! 💙

November 14 is Unstoppably Bold Day — a day for our CFC community to show up, stand out, and raise awareness for CFC syndrome around the world.

🌎 Are you hosting an awareness event on November 14? We want to hear from you!

Whether you’re organizing something at a school, workplace, community center, local business, or right in your own neighborhood, ROLL CALL in the comments! 👇

📍 Tell us your city, state/province, and country
💙 Share what you’re planning
🙌 Tag friends and family who are joining you

We want to see just how far our CFC community can reach on November 14.

Together, we are UNSTOPPABLY BOLD for CFC syndrome. 💙

09/03/2026

Join CFC International for an important conversation about mortality in Cardio-Facio-Cutaneous (CFC) syndrome and what current research can teach us about supporting individuals and families in our community.

CFC Syndrome and Mortality: Insights from the Latest Research
📅 Wednesday, September 23, 2026
⏰ 7:00 p.m. ET | 6:00 p.m. CT

We are honored to welcome Jacquelyn Schatzman Raposo, MS, CGC, Certified Genetic Counselor and Genetic Counseling Research Fellow at Baylor College of Medicine and Texas Children’s Hospital. She will be joined by Pilar L. Magoulas, MS, CGC, Certified Genetic Counselor, Chief of the Division of Genetic Counseling and Associate Professor at Baylor College of Medicine for Q&A. Pilar served as chair for Jackie’s master’s thesis research characterizing mortality in CFC syndrome.

Together, they will discuss research that included surveying bereaved CFC families, reviewing medical records, and scanning the published literature to better understand causes and contributors to mortality in CFC syndrome. The webinar will explore key findings, important takeaways, and recommendations for families based on the research.

💙 This webinar will be recorded for those who are unable to attend live.

Register here:
https://www.cfcsyndrome.org/patient-webinars

Open to families, caregivers, professionals, researchers, and others in the CFC community.

Advancing understanding and treatment for CFC syndrome.

09/01/2026

🏁 Start your engines... the countdown is ON! 🏁

In just 10 months, the CFC community will race into Indianapolis for the 2027 CFC International Family Medical Conference: Accelerating Hope for CFC Syndrome!

For families living with CFC syndrome, this isn't just another stop on the calendar—it's our pit stop for hope. It's where families reconnect, researchers share groundbreaking discoveries, clinicians learn from one another, and together we fuel the future of CFC syndrome.

We'll be counting down to an unforgettable week filled with:
🏎️ Life-changing medical education
💙 Meaningful family connections
🧬 The latest CFC research
🎉 Fun, laughter, and memories that last a lifetime

Every breakthrough begins with momentum. Every family adds horsepower to our mission. And every mile we travel together brings us closer to better treatments.
So buckle up... because we're officially ONE LAP CLOSER to the green flag!
📍 CFC International Family Medical Conference: Accelerating Hope for CFC Syndrome
📅 July 14–16, 2027
🏁 Indianapolis, Indiana
Hyatt Regency Indianapolis

Registration opens January 1, 2027. Scholarship applications open October 1, 2026. All details will be available here, via email, in the newsletters, etc.

Sponsorships are now open! Email Tuesdi Dyer at [email protected] for a sponsorship packet and more info.

08/25/2026

❤️ The 3rd Annual Harleybug Ride was a huge success! ❤️
What an incredible way to honor the life and memory of sweet Harley Melvin.

Harley lived with CFC syndrome and fought bravely every day of her life before passing away at just 10 years old. Each year, her family and friends come together for the Harleybug Ride—turning their love for Harley into something that continues to make a difference for other families affected by CFC syndrome. After all donations were received, the 3rd Annual Harleybug Ride raised $2,300! 💙

From riding ATVs and getting very, very muddy to selling memorial shirts and hats, cooking food, sponsoring activities, and simply showing up to support this family, so many people helped make the day special.

For CFC International, events like these mean so much more than a dollar amount. They help us continue connecting families, providing education and support, advancing research, and building a stronger future for everyone affected by CFC syndrome. Most importantly, they remind us that the lives of our CFC loved ones have an extraordinary impact on those around them.

Harley, your legacy rides on. ❤️

To Harley's family and the entire Harleybug Ride community: thank you for carrying Harley's memory forward and for choosing CFC International as the beneficiary of this beautiful tradition. We are deeply grateful!

08/19/2026

CFC International's New Family Orientations are a transformative webinar for caregivers and parents of adults and children newly diagnosed with CFC syndrome. We hope you will join us for our next New Parent and Caregiver Orientation this September 17th. We will guide your journey of care for CFC syndrome and provide a deeper understanding of the services CFC International provides.

You must register to attend:
https://us06web.zoom.us/meeting/register/y22rhC2ZR76JlNHVn9hU-w

After registering, you will receive a confirmation email containing information about joining the meeting. This session will not be recorded, to protect the privacy of our participants.

08/06/2026

Every breakthrough for a rare disease begins with a community that refuses to give up.

At CFC International, we're doing more than raising awareness—we're changing what's possible for individuals and families living with Cardio-Facio-Cutaneous (CFC) syndrome.

💙 We connect families so no one faces this journey alone.
🔬 We accelerate research that brings hope for better treatments.
🩺 We support multidisciplinary clinics that improve lives.
🎓 We educate healthcare professionals to improve diagnosis and care.
🤝 We bring together researchers, clinicians, industry partners, and families from around the world to move science forward—together.
🌎 We advocate every day for a future where every person with CFC syndrome has access to the care, resources, and opportunities they deserve.

Behind every program is a child learning to communicate, a parent finding hope, a researcher making a discovery, and a community that believes every life matters.

Because when we work together, hope becomes progress—and progress changes lives.

💙 Thank you to every family, donor, volunteer, researcher, clinician, and partner who makes this mission possible.

07/13/2026

How many people have CFC syndrome? The answer may surprise you.

If you've searched online for information about Cardio-Facio-Cutaneous (CFC) syndrome, you may have seen websites stating that there are only 200–300 people worldwide living with CFC syndrome.

That information is outdated.

Today, CFC International has more than 1,200 registered individuals with CFC syndrome from around the world, and we know there are hundreds more families who have never registered with our organization.

Even more importantly, emerging research suggests that the incidence of CFC syndrome may be as high as 1 in 55,000 people—meaning CFC syndrome may be more common than previously believed.

As our understanding of this rare condition continues to grow, it's more important than ever that families, healthcare providers, researchers, and advocates have access to the most current and accurate information.

That's why CFC International is committed to:
🩵 Sharing evidence-based information
🩵 Connecting families around the world
🩵 Supporting groundbreaking research
🩵 Ensuring the voices of our community help shape the future of CFC syndrome care

When you're looking for information about CFC syndrome, make sure you're relying on trusted, up-to-date sources.

🌐 Learn more at: www.cfcsyndrome.org

Together, we're building a stronger, more connected CFC community—and helping ensure that every family has access to the information they need.

06/24/2026

🌟 A heartfelt thank you to the Garabedien family for their incredible support in raising over $20,000 for CFC International! 🎉

With these donations, we're funding vital research and programming that will benefit current and future generations of children with CFC syndrome.

Gracie was diagnosed at just 4 months old, and the Garbediens are still learning to navigate this journey. They know they are supported by amazing teams, including clinicians at Boston Children's RAS Clinic, their community, and the more than 1,200 families registered at CFC International who have started this journey before them! 💖

Through JustGiving, special events, and Facebook giving campaigns, our communities are making a difference! 💪❤️

06/22/2026

🎉🎉It's always so amazing to watch the astounding accomplishments of the CFC community! Way to go Josh! You have a community of thousands rooting for you!🎉

Josh Follen will represent Team Wisconsin as their lone male powerlifter at the Special Olympics USA Games, competing in three events after a lifetime of beating the odds.