ProjectCASK

An innovative rare disease non-profit driving breakthroughs in treatments and a cure for CASK gene disorders.

[ProjectCASK @projectCASK]

CASK gene disorder is so rare that even experienced neurologists may never have seen it.
Signs include microcephaly, low muscle tone, epilepsy, delayed development, limited speech, and feeding challenges. If this sounds familiar, visit https://t.co/z93TxfSdIR

[ProjectCASK @projectCASK]

We're thrilled to announce our Family Day keynote speaker: @jessronne !
Author, caregiver advocate, founder of @TheLucasProject, and host of Coffee with Caregivers. Mom of 8, including her son Lucas, who has profound disabilities.
November 12. Houston, TX https://t.co/33pdNpU2RP

[ProjectCASK @projectCASK]

Our families are rallying. Your employer can too 🦁⚡️Roar 2026 already has its first Gold $10k sponsor! Know a company that belongs in this room? We have a sponsorship package ready to share. Email [email protected] to get started.
🔗 https://t.co/33pdNpU2RP

[ProjectCASK @projectCASK]

Registration is officially OPEN for Roar 2026: The CASK Conference! The first-ever in-person scientific and family conference for the CASK gene disorder community. Nov 10–12, Houston, TX. Early bird pricing through September 30. Details:
🔗 https://t.co/33pdNpU2RP

[ProjectCASK @projectCASK]

In 2025, something shifted. 11 studies. Multiple continents. Families, researchers, donors, and coordinated work across models, data, and strategy. In 2025, the science for CASK gene disorder began to move as one. Read the full impact report: https://t.co/PHcrFLa762 #CASKgene

[ProjectCASK @projectCASK]

Introducing Project CASK Canada — our first global affiliate. One SAB. One Roadmap. One community that refuses to wait. For every Liocorn, everywhere. Interested in affiliating? [email protected]

[ProjectCASK @projectCASK]

Today is #PurpleDay 💜 For families with CASK gene disorders, epilepsy awareness is a 365-day journey. We stand with our community in the fight for research & a healthy future.

Take action: Please consider joining the CASK epilepsy study:
https://t.co/AVq42uSRIY

[ProjectCASK @projectCASK]

ROAR Episode 12: Why multiple treatments for cerebellar degeneration in CASK? It’s not one broken pathway but a chain of disruptions, creating multiple chances to intervene from early to later stages. A key insight into how research into CASK is evolving. https://t.co/HnfB5eKNvE

[ProjectCASK @projectCASK]

@RareRevolutionM We can’t thank you enough @RareRevolutionM for giving our children and our mission a platform to amplify our advocation! It has been a delight and an honor.

[RARE Revolution Magazine® @RareRevolutionM]

Ultra-rare doesn’t mean fragmented.

Our Liocorn Roster connects 550+ people with CASK across 57 countries—turning data and community into an investable foundation for therapies. @ProjectCASK

[RARE Revolution Magazine® @RareRevolutionM]

Thank you to RARE Revolution Magazine for opening your platform to rare disease communities and for letting us share our story this Takeover Tuesday.
Today, because of families and scientists working together:
There is a growing research field.
here are global collaborations.
There are preclinical therapies in development.

And there is a community proving that rigor, collaboration, and persistence can change what’s possible.
If this story resonates, join us on February 21 for Rhythms for Rare — a global virtual concert bringing our community together to fuel the next chapter of CASK gene research.
We are Project CASK.
And this is how the future of CASK gene disorders is being built.
@ProjectCASK

#ProjectCASK #CASKResearch #RareDisease #CASKgene #GeneTherapy

[RARE Revolution Magazine® @RareRevolutionM]

To treat CASK we must de-risk the science. @ProjectCASK is joining forces w/ @CPathInstitute’s TRxA to co-fund CASK translational studies. Providing researchers w/ funding & regulatory support to translate CASK science into treatments. Researchers: https://t.co/WV5v7wITh5

[RARE Revolution Magazine® @RareRevolutionM]

In 2024, @ProjectCASK deployed $375k to Dr. @MingshanXue to de-risk gene therapy. He delivered data that named him a 2025 Oxford-Harrington Scholar @OHRareDisease This is leverage. We don’t wait for the system to save us. We fund the science that makes treatment inevitable.

[RARE Revolution Magazine® @RareRevolutionM]

Some call our children unicorns—rare & magical. But facing this disorder takes the grit of a lion. Enter the Liocorn. Rare as unicorns. Strong as lions. This is the spirit of @ProjectCASK. We honor their tenacity by fighting for their future. Read stories: https://t.co/PAHQMPhAfI

[RARE Revolution Magazine® @RareRevolutionM]

Developing treatments for rare disorders takes an ecosystem. @ProjectCASK is the largest global funder of CASK research, advancing models, biomarkers, multiple therapeutic strategies, & mapping 550+ patients. We don't wait for answers. We convene, connect & accelerate the science

[RARE Revolution Magazine® @RareRevolutionM]

CASK is a critical gene for brain development. Located on the X-chromosome, it has multiple roles before, in & post-synapse. CASK mutations can affect all aspects of human development & health @ProjectCask is driving research to restore those connections https://t.co/2IhkSfoPar

[RARE Revolution Magazine® @RareRevolutionM]

Hello, we are @ProjectCASK, honored to join @RareRevolutionMag for Tuesday Takeover. Our story starts where medicine didn’t have answers, so parents built them. We are home of the Liocorn—rare as a unicorn, fierce as a lion. Join us as we drive CASK science from insight to cure.

[ProjectCASK @projectCASK]

📣 New funding pathway for CASK treatments. Project CASK is partnering w/ @CPathInstitute to co-fund translational studies thru the BRIDGe 2026 prgm; dedicated funding & regulatory support to translate CASK science into treatments Pre-proposal due March 16 https://t.co/6b8e2Mlzz2

[Critical Path Institute (C-Path) @CPathInstitute]

We are pleased to invite you to register now for Critical Path Institute's 2026 COA Program Annual Meeting, taking place April 16–17, 2026, at the Washington Marriott at Metro Center in Washington, D.C.

Register now: https://t.co/NODfEOSDOI

[Becky Quick @BeckyQuick]

Sharing our family’s story this week. Thanks to @Kramer_Katie for her beautiful work on this piece. ❤️
https://t.co/ECOkRYMEpF