Tag Archive for: ion283

March 10, 2026 Statement from Fondazione Telethon

Telethon’s Statement on the ION283 Clinical Trial Pathway for Lafora

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Fondazione Telethon Logo

Fondazione 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.

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!
Noventia Pharma logo

Noventia’s Reply to Lafora Patient Community

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Noventia replied to the open letter from the Lafora patient community that they were in the final stages of formalizing an agreement with Fondazione Telethon for the clinical development of ION283. Here are their answers to some of our questions:

“• Clinical Trial Phase: We are preparing to submit an application for a Phase 1 clinical study to the regulatory authorities by the end of this year. This study will primarily focus on safety, but will also include preliminary efficacy endpoints, and is expected to enroll 20 patients.

• Trial Sites: The coordinating center identified for the study is the IRCCS Institute of Neurological Sciences in Bologna, Italy, a center of excellence in this field. Should there be any delays in certification, we have contingency plans to involve one or more additional European centers to avoid unnecessary delays.

Regarding your question on site accreditation, in Italy, Phase 1 clinical centers must be certified under the specific AIFA regulation 809/2015. Our selected center in Bologna is in the process of obtaining this certification, which we anticipate will be finalized by the time of the study submission. For any potential international sites, while this Italian regulation does not apply, we will ensure they meet comparable high-quality standards. The thorough evaluation and selection of all clinical sites will be conducted as soon as the final protocol is ready.

• Transparency and Communication: We are committed to keeping the patient community updated. Once the clinical protocol is finalized, we will organize a dedicated meeting with patient representatives and associations to share all relevant details and answer your questions. Up to now, the Italian patient associations AILA and Tempo Zero have been regularly consulted and kept aligned through periodic meetings and updates. We will continue this approach, ensuring that all families remain informed as we move forward.”