About the Organization

The Mission

The mission of Chelsea’s Hope is to improve the lives of those affected by Lafora disease and help accelerate the development of treatments.

The Challenge

The problem we aim to address is improving outcomes for Lafora disease. It is a rare neurodegenerative condition affecting one in 10 million children. Symptoms include epilepsy, ataxia, childhood dementia, cognitive decline, and difficulty speaking, walking, and eating. There is currently no cure; the disease is always fatal.

An infographic that includes half a circle of Lafora Disease in the center and half a circle beneath with text that says 'What is Lafora disease? Also called Lafora progressive myoclonus epilepy, it's a rare neurodegenerative glycogen storage disease that is terminal.' Around the circle, symbols and text explain more about the disease. The text says 'childhood dementia, palliative care to treat symptoms, increasingly severe seizures, affects an estimated 200 people worldwide, genetic condition, no cure, healthy children develop symptoms around puberty, ataxia: loss of muscle control.

Lafora Disease research has little to no funding from the Federal Government. This is due in part to Lafora’s classification as an orphan disease (a condition that affects fewer than 200,000 people nationwide). The other challenge is funding for Lafora research is in competition with more than 7,000 other orphan diseases. There simply isn’t enough funding to go around. Our hope is that by raising awareness about Lafora Disease, we can dramatically increase the funding for research needed to find a cure. chelsea hope

Our Impact

We are proud to be the first Lafora disease advocacy group that brought together families from around the world. We also partner directly with biotech and industry partners to advance treatments.

Accomplishments:

Raised $5.5 million for Lafora disease research since 2007.
Hosted and co-hosted the annual international Lafora Disease Science Symposiums.
Fostered the Lafora Disease Families Facebook group since 2020.
Assisted with recruitment for a natural history study in 2019.
Received a Chan Zuckerberg Initiative Rare As One grant.
Received a 2022 #RAREis Global Advocate Grant from Horizon Therapeutics.
Facilitated the Lafora Disease Research Roundtables since 2023.
Received a 2023 Patient-Partnered Collaborations for Rare Neurodegenerative Disease RFA.
Coordinated the application for ICD-10 Codes for Lafora disease. Lafora disease was awarded the unique code G40.C in 2023.
Chelsea’s Hope engages an audience of 2k+ through social media networking to connect with the community and raise awareness. Since 2007, we have shared patient and family stories on our website and news media to educate about Lafora disease and fundraise for research.

What’s next:

2024 Research Roundtables.
2024 Lafora Disease Science Symposium.
Coordinating discussions and funding a consensus document for biomarkers and treatment of Lafora disease.

We invite you to read Chelsea’s story and to meet the other Children of Chelsea’s Hope. Then, please donate as much as you can to help stop this deadly disease. chelsea hope