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

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.