August is here — and so is Make a Splash for CASK, our annual global fundraiser to raise awareness and fuel therapeutic development for CASK gene disorders.

In this episode of The ROAR, Project CASK co-founder Hitomi Kubo invites listeners behind the scenes to learn how research, community, and creativity come together in our mission to find treatments and a cure for CASK. Using a memorable banana split ice cream sundae metaphor, Hitomi walks through the essential ingredients needed to build a successful rare disease nonprofit — and the powerful impact of community-led fundraising.

You’ll learn:

• Why deep understanding of CASK gene disorders is essential to progress — including the world’s first CASK prevalence study, community-reported symptom data, and strategic scientific collaboration.

• How Project CASK is building research infrastructure — from mouse models and brain organoids to a biorepository of CASK samples.

• What cutting-edge therapeutic programs we’re funding — including gene replacement therapy, small molecule strategies targeting cerebellum degeneration, and an in-depth epilepsy study.

• Why education and outreach matter — and how we’re equipping families, clinicians, and the public with tools to drive awareness and advocacy.

• How your participation in Make a Splash for CASK powers every part of this mission — and why every single fundraiser makes a difference.

Whether you're a parent, researcher, or advocate, this episode will inspire you to get involved — and give you the roadmap for how community engagement translates into real progress for ultra-rare CASK disorders.

Join the Movement:
Make a Splash for CASK this August by signing up: givebutter.com/Liocorn/join
Learn more: projectcask.org
Follow us on social: @projectcask

🎙️The latest episode of the ROAR is here, full of exciting updates and real ways you can help move the needle for the CASK community.

https://youtu.be/X3DoyBRXUoU

Here’s what’s inside:

🧬 What recent progress in gene replacement trials means for our CASK kids and why it matters

https://youtu.be/_wZ3-GUuyBc?si=A6ZcqCiNKCgj7yS2

🧠 A spotlight on the 2nd CASK Scientific meeting - collaboration for acceleration plus a deeper look at cerebellar degeneration

🧪 How our sweet CASKies can participate in sample collection for the CASK biorepository hosted by COMBINEDBrain and the biomarker study

https://www.projectcask.org/cask-assets

💛 Why developing therapies takes more than just funding studies and what we all can do

https://www.citizen.health/partners/project-cask

🌊 How families can Make a Splash for CASK this August to advance breakthroughs our kids urgently need

https://givebutter.com/Liocorn/join

🗣️ Why now’s the time to contact your senators and help protect Medicaid for rare and medically complex kids

https://everylifefoundation.org/rare-advocates/take-action/

🗓️ And how we’re building toward CASK Gene Awareness Day on July 15, a moment to raise our voices together

https://www.twibbonize.com/profile

Get ready, get set and ROAR! We may be rare, but together we’re loud, loving, and unstoppable.

🎙️ Listen here: https://youtu.be/X3DoyBRXUoU

The latest episode of [ The Roar ] is here!

This episode, we’re diving into:

🩵💜🩷 A huge thank you to our community for your incredible Rare Disease Day advocacy—your voices matter!

🔬 [1:00] Research updates - what’s happening behind the scenes on CASK gene research

🎨 [5:15] The Ultra Rare Collection fundraiser – Join us, share and follow

@ultrararecollection

⚖️ [7:35] Federal budget cuts & advocacy for Medicaid – Sharing our stories to help build understanding of the importance of Medicaid, the Everylife Foundation for Rare Diseases, and Project CASK-funded research is still moving forward!

+ Medicaid and Federal Funding take action with Everylife Foundation:

https://everylifefoundation.org/rare-advocates/take-action/

+ Sign up for Citizen Health + Project CASK:

https://www.citizen.health/partners/project-cask

+ Create your personalized Liocorn Donation page:

https://givebutter.com/Liocorn/join

+ Register with Project CASK:

https://www.projectcask.org/register