Updates for the Lafora Therapy Pipeline: Requesting information from our Research Community

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At our 2025 Lafora Symposium, several of the Lafora patient organizations decided to work together to better support therapy development for Lafora disease.

To help us prioritize therapies to support, we are requesting periodic updates on novel and repurposed therapies in development for Lafora. If you are a clinician or researcher working on a Lafora Therapy, please complete this form so that we can educate families about your research and determine the best way to support the development of your therapy.

Submissions will be reviewed by A.I.L.A., Cel-Luz Association, Chelsea’s Hope, France Lafora,  and TempoZero.

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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
Noventia Pharma logoNoventia’s Reply to Lafora Patient Community