text says "social security" on a piece of paper in a typewriter

Social Security Added Lafora to Compassionate Allowances List

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U.S. Lafora patients can qualify for disability benefits faster through the Compassionate Allowances List. In fact, SSA says your application would be “moved to the front of the line.”

The SSA reported that Lafora is now one of 314 serious medical conditions on the list, and “since the initiative started, over 1.2 million people have been approved.” This action should speed up the process for Lafora families seeking disability benefits.

We hope this decision helps families around the world access similar government support. Chelsea’s Hope anticipates more funding opportunities for the Lafora disease community to advance research, potential treatments, and improve patient care. We are grateful that the SSA recognizes the urgency we face.

Elpida Therapeutics’ Press Release

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Elpida Therapeutics released a press release about acquiring ION283 and advancing this potential therapy. We are fortunate that Terry shared a letter with us earlier in the week.

For more information about the nonprofit biotech company and the license transfer from Ionis, you can read the press release that Elpida Therapeutics sent out here.

Read Press Release

Please direct any questions about ION283, Elpida Therapeutics, or the Safety Study to ion283@chelseashope.org.

August 3, 2026 Letter from Terry Elpida Therapeutics

ION283 to be Developed by Elpida Therapeutics

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Exciting news for the Lafora community! 

Ionis reached an agreement for the future of the ION283 asset with Elpida Therapeutics. Terry Pirovolakis, CEO of Elpida Therapeutics, asked us to share this letter with the Lafora community to introduce their company and their plans for ION283. You can read the letter here: 

Read Letter

Chelsea’s Hope is committed to advocating for the Lafora community and helping accelerate the development of this potential treatment. Please direct any questions about ION283, Elpida Therapeutics, or the Safety Study to ion283@chelseashope.org, and we will get back to you.

As a reminder, Chelsea’s Hope will host a Lafora Therapy Q&A on August 11 at 1:30 PM EDT. This session will go over new resources we’ve recently shared about the Lafora therapeutic pipeline. Then, we will answer your questions submitted in advance of the meeting, ending with some time for questions and responses. We will use TransPerfect AI translation for captions. Please register in advance here.

Update on the ION283 Program from Telethon

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Below is an update from Fondazione Telethon, which shares that Ionis transferred development rights to another organization and reaffirms Telethon’s commitment to the Lafora community.

Dear Members of the International Lafora Patient Community,

Following our previous communications, we would like to provide an important update regarding the ION283 program.

We have been informed that Ionis has decided to transfer the development rights of ION283 to another organization, different from Fondazione Telethon. As this decision rests entirely with Ionis, it is not under our control, as we have consistently clarified in our previous updates.

From the outset, our only priority has been — and will remain — to ensure that people living with Lafora disease have a real opportunity for a therapeutic option: in this spirit, we have donated all the work and materials developed over the past months to the organization identified to continue the program, in order to facilitate the fastest possible progress.

We are fully aware of the sense of urgency experienced by families and of how deeply time matters in the context of this disease. For this reason, we believe that enabling continuity — regardless of the actors involved — is the most responsible choice in the best interest of patients.

At the same time, we wish to reiterate Fondazione Telethon’s close commitment to the Lafora community: we will continue to support scientific research through the avenues that can best safeguard all patients, in full respect of internationally recognized standards, regulatory requirements, and the principles of scientific rigor and excellence.

Please be assured that we have done — and will continue to do — everything within our reach to support the Lafora community.

We remain close to you and committed to sharing further updates as soon as reliable information becomes available.

With respect and commitment,

Alessandra Camerini

Head of Relations with Patients and Patient Organisations