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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
ION283 Clinical Pathway Update January 8, 2026

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 – Fondazione Telethon

“Dear Members of the International Lafora Patient Community,

First and foremost, we would like to thank you for your continued engagement, commitment, and trust: we are fully aware of the urgency faced by families affected by Lafora disease and of how deeply time matters in the context of a rapidly progressive condition.

For this reason, Fondazione Telethon shares your determination to move forward as quickly as possible, while ensuring that every step is taken responsibly, transparently, and in the best interest of patients’ safety and future access to ION283 treatment.

In light of the current situation, we would like to share an update on our ongoing efforts.

At present, Fondazione Telethon is actively working to establish a direct dialogue with Ionis, with two key priorities:

    • accelerating the start of the clinical trial as much as possible, and
    • obtaining additional scientific and clinical information on the investigational drug that is not yet fully available to us and that is essential to plan the next steps in a solid and informed way.

Our intention is to facilitate a pathway that allows the clinical program to move forward efficiently, while ensuring that decisions are based on the most complete and up-to-date data available.

We remain fully committed to transparency and open communication: as soon as further information becomes available and concrete timelines can be defined, we will promptly share updates with the international patient associations and the broader Lafora community.

We recognize the weight of expectations, concerns, and hopes carried by families every day: please be assured that Fondazione Telethon is working with the utmost sense of responsibility and urgency to help transform these hopes into a concrete and accessible clinical pathway.

Thank you for your continued collaboration and trust.”

We share your frustration at this setback. As we learn more information from partner organizations, we will share it.
We are very grateful that Fondazione Telethon has made itself available to step up to help develop the drug, as well as being so communicative with our community. Let’s remain united and hopeful!
Lafora Therapy Town Hall Join us to discuss future treatment options and plan as a community. 28 october 2025 tuesday Register online to attend the Zoom meeting.

Announcing Lafora Therapy Town Hall

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Lafora Therapy Town Hall Join us to discuss future treatment options and plan as a community. 28 october 2025 tuesday Register online to attend the Zoom meeting.

 

Questions about what therapies are next for the Lafora community? We are hosting a Lafora Therapy Town Hall to discuss what treatments the community can rally behind to bring from pre-clinical studies to clinical trials for our children.

Join us next Tuesday, October 28th, at 11 am PDT via Zoom.

As Lafora patient organizations – Chelsea’s Hope, Cel-luz, France Lafora, A.I.L.A. – we want to be united in our message and pursuit of treatment.

It’s been a worldwide effort to fund the safety study / phase 1 trial for ION283 at UTSW. We’ve been working towards it for years. If our patient community is going to bring another therapy forward, we must be unified in our next steps.

Families and caregivers, we hope you will be able to attend this event. Your voice matters!

Register here Right-open Right-open
The background is bright yellow. On the left is a child with blue face paint. To the right is another child, who has red, white, and black face paint on, Between the children, the text reads, 'FACE it To make childhood dementia impossible to ignore'.

FACE It to Raise Awareness of Childhood Dementia

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Chelsea’s Hope joins the Childhood Dementia Initiative (CDI) in recognizing next Wednesday, September 17, 2025, as Childhood Dementia Day. We will join the FACE It awareness campaign to make childhood dementia impossible to ignore. 2025 is the third year that Chelsea’s Hope is participating in the movement. We invite you to join us!

The background is bright yellow. On the left is a child with blue face paint. To the right is another child, who has red, white, and black face paint on, Between the children, the text reads, 'FACE it To make childhood dementia impossible to ignore'.

Did you know that Lafora is one of hundreds of genetic conditions that cause childhood dementia? It is devastating. We need more attention and support to reach treatments, improve outcomes, and save lives.
That’s why we’re joining the Childhood Dementia Initiative‘s #FaceIt campaign to spread awareness that children get dementia, too.

View a childhood dementia fact sheet HERE.

Join us in a casual Zoom on September 17th from 9-10 AM PDT and help us raise awareness of childhood dementia. We invite you to join the Zoom call with your face colorful and impossible to ignore, or paint it as your art activity during our virtual meet-up. At the end, we’ll take a group photo for anyone who wants to participate. Together, we’ll draw attention to childhood dementia.

I’m in!