Tag Archive for: ionis pharmaceuticals

January 20, 2026 Update Fondazione Telethon logo

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

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Fondazione Telethon Logo

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 in gaining the license for ION283, they plan to:

  • Start a clinical trial as soon as possible
  • Cover all the clinical costs of the trial
  • Allow European patients currently in the Safety Study to complete their visits in Italy and continue receiving the drug after the study concludes

We appreciate the clarifications from Telethon! We will continue to update the Lafora community as we learn of developments for ION283.

As a reminder, please email your questions or concerns to ion283@chelseashope.org, and we will advocate on your behalf.

Read the statement from Telethon
IONIS Statement for the Lafora Community September 4, 2024

Ionis Statement for the Lafora Community

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IONIS logoIonis shared a statement with Chelsea’s Hope for the Lafora community. They have agreed to license the ION283 program to Noventia Pharma, and Noventia will assume responsibility for developing it. 

The licensing of the ION283 program will not impact the Safety Study. That will continue as planned, and we will update our community as soon as enrollment is open on clinicaltrials.gov.

Read statement

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