The mission of Chelsea’s Hope is to improve the lives of those affected by Lafora disease and help accelerate the development of treatments.
Chelsea’s Hope Lafora Children Research Fund is an IRS 501(c)3 non-profit organization. EIN: 27-1008382
Chelsea’s Hope c/o Dr. Donohue
976 Maywick Dr.
Lexington, KY 40504
Telethon’s Statement on the ION283 Clinical Trial Pathway for Lafora
/by Chelsea's HopeFondazione 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 and in the United States, provided that the highest ethical and scientific standards are upheld and that any decision is taken exclusively in the best interest of patients.
Fondazione Telethon acknowledges the complexity of the ION283 case, also from the perspective of Ionis, which currently holds the license for the compound.
The essential authorizations required to set up a clinical study in Italy fall within Ionis’ decision-making space. Therefore, at this stage, Fondazione Telethon cannot serve as the reference point or source of information regarding a possible future development path for ION283. The decision concerning the continuation, redesign, or activation of a clinical program is not under our direct control. Should concrete and reliable updates become available, we will share them promptly.
We are aware that several research groups, in different Countries, are actively conducting studies on Lafora disease: this reflects the commitment of the international scientific community to advancing knowledge and identifying potential therapeutic strategies.
While dealing with the hard work to ensure progresses in identifying therapeutic solutions, we respectfully invite families, associations, and researchers to bear in mind that the scientific pathway required to transform science in therapies does not allow for shortcuts. The timelines necessary to ensure safety, efficacy and compliance with regulatory standards cannot be compressed beyond what scientific rigor and ethical standards allow.
We are aware that this message may sound demanding. However, clarity is necessary to preserve families who are already facing extraordinary challenges and a sense of unpostponable urgency every day.
Our commitment remains firm: to pursue research with responsibility, scientific soundness, transparency, and deep respect for patients and their loved ones.
As a reminder, please email your questions or concerns to ion283@chelseashope.org.
2025 Annual Report
/by Chelsea's HopeChelsea’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 for supporting Chelsea’s Hope last year. We appreciate your continued partnership to reach a cure!
Stay connected by signing up for our mailing list and following our social media for regular updates. We always welcome volunteers to support our cause and invite you to join Chelsea’s Champions with a monthly gift to support our work in 2026 and beyond.
Telethon’s Update on the ION283 Clinical Trial Pathway for Lafora Disease
/by Chelsea's HopeFondazione 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:
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.
Update on the ION283 Clinical Pathway
/by Chelsea's HopeWe 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:
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.”