Entries by Chelsea's Hope

Telethon’s Statement on the ION283 Clinical Trial Pathway for Lafora

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Fondazione Telethon has kindly provided Chelsea’s Hope and the other advocacy organizations with a statement for the Lafora patient community. At their request, we are sharing the communication below: Dear Members of the International Lafora Patient Community, Fondazione Telethon remains open to contributing to the development of ION283 and to collaborating with partners in Europe […]

2025 Annual Report

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Chelsea’s Hope published our 2025 Annual Report! Inside, you’ll find a message from the President of the Board of Directors, Jenifer Merriam, and a summary of our impact in 2025. We also include a financial statement, goals for 2026 and beyond, and acknowledgments. Thank you to our generous donors, committed research network, and courageous families […]

Telethon’s Update on the ION283 Clinical Trial Pathway for Lafora Disease

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Fondazione Telethon has kindly provided Lafora patient organizations with a statement that includes some clarifications about ION283. As they note, Telethon does not currently have a contract with IONIS. Until they have a contract for the license, they have no authority to submit a protocol to the EMA/FDA for a trial. If they are successful […]

Update on the ION283 Clinical Pathway

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We understand many families are wondering what’s next for the development of the ION283 drug. Unfortunately, we received an update today from A.I.L.A. and Fondazione Telethon that Noventia Pharma returned the license for ION283 and will no longer be bringing it to a trial. Update on the ION283 Clinical Trial Pathway for Lafora Disease – […]