CHAMP1 UK

05/06/2026

⏰ 3 MONTHS TO GO! ⏰
Can you believe it? We are just three months away from the CHAMP1 Research Alliance Family & Scientific Engagement Conference 2026! 💜🧬

If you haven't registered yet, now is the time. We cannot wait to welcome our incredible global community to the Children's Hospital of Philadelphia (CHOP) from Thursday, 3rd September through Sunday, 6th September.

Whether you're coming for the latest research updates, the family sessions, or simply to be in the same room as people who truly understand your journey — this is a weekend you will not want to miss. ✨

Here's what's waiting for you in Philadelphia:

🔬 A dedicated scientific symposium with cutting-edge CHAMP1 research
👨‍👩‍👧 Family-focused presentations and a community outing
🏨 Preferential rates at the Sheraton Philadelphia University City Hotel
💜 Attendance Support Grants available for families who need help with travel and accommodation costs

📝 Register, apply for a grant, and find the hotel booking link at: https://chmp.one/conference

Philadelphia is calling. We'll see you there! 🧬💜

CHAMP1Research

04/05/2026

Happy Birthday to the "Father of CHAMP1," Dr. Kozo Tanaka! 🎂

Today we celebrate the man who started it all. Dr. Tanaka’s team first identified and named the protein that defines our community.

Dr. Tanaka, thank you for your unwavering dedication to our CHAMP1ONs. From your initial discovery to your ongoing work, you have given our families the gift of understanding and the hope for future treatments. 💜

02/05/2026

Join us in wishing our CHAMP1ON Sophie a very happy 9th birthday 🎁🎈🎂
We hope you have an amazing day 💜

29/04/2026

A great article about Stacey and Calum.
They were one of our first CHAMP families that helped create our amazing community and welcomed many of us after we received our diagnoses 💜

Stacey McPherson's son Calum, 19, is non-verbal after he was born with a rare gene mutation known as CHAMP1.

21/04/2026

Join us in wishing our CHAMP1ON Alex a very happy 21st birthday! 🎉🎈🎁

We hope you’ve had an amazing day 💜

10/04/2026

We are incredibly excited to announce that the CHAMP1 Research Alliance has been awarded a $100,000 grant from the RTW Foundation.

This funding will strengthen our ongoing partnership with the Berto Lab at the Medical University of South Carolina (MUSC). Together, we are focusing on ground-breaking work to develop RNA therapies for CHAMP1-related disorders.

Specifically, this grant will accelerate our project exploring the use of Target Site Blockers (TSBs). TSBs are a cutting-edge form of Antisense Oligonucleotides (ASOs) designed to instruct the body to restore normal CHAMP1 protein levels and improve neuronal function.

This project is a critical step forward for all individuals with CHAMP1.
At a minimum, this research will help us better understand how different CHAMP1 mutations behave (including dominant-negative effects), which is vital for developing future precision treatments.

We are so proud to drive this innovative research and to continue fighting for the best quality of life for our CHAMP1ons. A huge thank you to the RTW Foundation for partnering with us to make the impossible happen!

09/04/2026

Happy birthday to our CHAMP1ON Laura who turned 2 yesterday 🎉🎁🎈
We hope you had a fantastic day 💜

09/04/2026

Join us in wishing our CHAMP1ON Sienna a very happy 7th Birthday 🎁🎈🎉
We hope you have an amazing day 💜

02/04/2026

We are thrilled to announce that our CHAMP1 Gene Guide developed in partnership with Unique - Rare Chromosome Disorder Support Group is now available in French.
🇫🇷 https://chmp.one/guide-fr
Thank you to those who helped translate and review the guide.

This joins the English and the Spanish version that have already been published, with a number of other translations underway.
🇺🇸🇬🇧 https://chmp.one/guide
🇪🇸 https://chmp.one/guide-es

01/04/2026

To show their support, on March 20th the teams at Charles River Laboratories Évreux, including Noam's parents came together for a group photo in purple for our CHAMP1ONs💜

Thank you for supporting our CHAMP1ONs on CHAMP1 Awareness Day!