Creating Awareness,
Connecting Families,
Funding Research,
Maintaining Hope.

In Memoriam
Chelsea Gerber
1990-2016

Creating Awareness,
Connecting Families,
Funding Research,
Maintaining Hope.

Lafora patient
Adela Richer

Creating Awareness,
Connecting Families,
Funding Research,
Maintaining Hope.

In Memoriam
Kelsey Anne Harris
1995-2014

Fund treatment for Lafora disease
Link to: Children of Chelsea’s Hope

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Meet

The Children of Chelseas’s Hope

Link to: Newly Diagnosed

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Link to: Donate

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Watch Our Stories

Fighting the Rare

A documentary about Lafora disease that features Chelsea’s Hope.

Living with Lafora

The Merriam family tells their story after a devastating diagnosis.

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What’s New?

Dear Noventia Pharma Team, We are families affected by Lafora disease who are holding on to hope through your work. Your dedication to developing a treatment for Lafora means everything to us. We were excited when you shared the news of the ION283 license purchase and your intentions to create a clinical trial last year, but we are anxiously awaiting more details. We know that any future progress with ION283 will come from your company, so we kindly request a public update on what is next for the drug’s development after the safety study at UTSW. As you know, the current ION283 safety study includes only 10 children. While we are grateful this study is happening, many more families were heartbroken to learn our children could not participate. These families are now left waiting, watching their children’s condition progress, and praying for the next opportunity. We are counting on Noventia to help make that opportunity possible. For many of us, your work is the only hope we have left. Our children do not have time to wait. Lafora disease is aggressive and unforgiving—we are pleading for a chance to save our children’s lives. We desperately urge Noventia to share your plans and move forward quickly with a clinical trial or expanded access program so more children can receive this potentially life-saving treatment. You have our full support in this mission, and we are willing to assist however we can.

Open Letter to Noventia

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Zaina Zaid Nemer

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Town hall To discuss fundraising efforts and plan as a community. FULLY FUND THE SAFETY STUDY21 MAY 2025 WEDNESDAY Southwestern William P Clements Jr. U Register online to attend the Zoom meeting.

Announcing a Town Hall: Fully Funding the Safety Study

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Partners

AEVEL Asociacion Espanola para vencer a la enfermedad de lafora
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Rare As One Chan Zuckerberg Initiative Proud member of the Rare As One Network
Rare Epilepsy Network Proud Member
NORD 2025 Member Platinum
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