Recognition of a Historic Milestone in Lafora Disease Research

With immense joy and deep gratitude, we are about to announce a long-awaited milestone, marked by ardent hope and, at times, by unsettling anxiety. Tempozero wishes to express a heartfelt and respectful recognition to Dr. Berge Minassian for his tireless commitment and dedication to the research and development of the ASO (antisense oligonucleotide) drug over the past thirty years.

Dr. Minassian, supported by a team of exceptional professionals and clinicians worldwide, has achieved a milestone that once seemed unattainable. The results obtained in the preclinical phase have demonstrated not only the safety of the drug but also an efficacy that makes us even more confident. He has brought a new ray of hope to all the families involved in this challenge.

Today, with the ION283 drug ready to enter Phase 1 clinical trials, we can finally say that we are one step closer to realizing a dream that has accompanied us for years. The faint hope that has guided our actions is transforming into a solid certainty.

However, it is crucial to emphasize that, to reach this ambitious milestone, the first ten patients will face significant costs to travel to UT Southwestern Medical Center in Dallas every three months for a period of two years. This investment, totaling $1.5 million, will cover only the hospitalization expenses of our beloved young children. In addition to these costs, families will also have to bear the expenses necessary to ensure all transfers, along with food and lodging.

At this critical moment, we turn to you for an additional effort and renewed demonstration of solidarity. Your generosity and support are now more crucial than ever, as we get closer and closer to achieving our collective goal.

Therefore, I wish to express my most sincere thanks, on behalf of all of us, to Dr. Minassian for his unwavering dedication and for guiding us toward this extraordinary milestone. His passion and commitment have ignited a new and vibrant hope for all the families affected by the devastating Lafora disease.

Together with TempoZero, Chelsea’s Hope and Lafora France have also demonstrated their commitment on a global level by launching a massive fundraising campaign, thus supporting this vital cause. The union of these forces will enable us to provide a concrete response to the needs of patients and their families, while continuing to promote research and development.

Sincerely,

Tullio Bressanello

President of TempoZero