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.