Fund treatment. Save lives. Change the future.

Participating in the ION283 Safety Study is a life-changing opportunity for 10 Lafora patients. It gives the worldwide community hope that we can find treatments for all our kids.

The only barrier is funding. We have to reach $1.5 million to fully fund the clinical costs of the study.*

ION283 could halt the progression of Lafora, a rare, fatal disease that causes epilepsy and childhood dementia. It was successful in preclinical studies and our best chance to save children today. We need a cure, and this is a huge step forward.

As we are updating this in October 2025, all 10 patients are enrolled. It's thanks to your generosity over the past year that Chelsea's Hope has been able to support the study.

However, we are still fundraising because we MUST ensure all 10 patients can complete all their hospital visits. The study must run its course!

Donate today!

Your contribution will go directly to the clinical costs of the study. Not only are the enrolled Lafora patients finally trialing a treatment option, but if successful, this small study can grow into a larger trial or lead to compassionate use of the drug for other patients.

Join us today in our efforts to raise awareness about Lafora disease and funds for a cure. Spread the word—time is of the essence, and everyone can make a difference.

You can learn more about the ION283 Safety Study HERE.

Chelsea's Hope Lafora Children Research Fund fights Lafora disease, a rare, fatal, neurodegenerative disorder that affects children. Symptoms include myoclonus, seizures, childhood dementia, ataxia, difficulty walking, talking, and eating. Our mission is to improve the lives of those affected by Lafora and help accelerate the development of treatments. Simply put, we want to save our kids!

Learn more about Chelsea's Hope. >

*Note: we will change our fundraising goal according to the amount needed to fund the study fully. After all the fundraising the Lafora organizations and community leaders have done, we still have $153k that we need to raise/pledge.

Fully fund the ION283 Safety Study

Fund treatment for Lafora disease!

Team Leaderboard

Team Members