Foundation Attends n-Lorem Foundation’s Third Annual Nano-rare Patient Colloquium
October 27, 2025
Nano-rare disease advocates, clinicians, researchers, and industry collaborators gathered in Cambridge (MA) earlier this month for the n-Lorem Foundation’s 2025 Nano-rare Patient Colloquium.
For nano-rare APBD patients with the deep intronic variant, the n-Lorem Foundation’s work to develop personalized medicines -- using antisense oligonucleotide (ASO) technology -- is a critical therapy development program
Two sessions of interest included:
Dr. Konstantina Skourti-Stahaki (Senior Director of Discovery and Research) discussed the ASO scientific discovery process. Her presentation used a published research study about the ASO which targets the GBE1 gene. You can read a summary of the study here!
Andrew Serrano (Digital Marketing Manager) shared the “Patient Empowerment Podcast” and other resources that n-Lorem has developed to support nano-rare patients.
For all of the conference recordings, be sure to visit the Nano-rare Patient Colloquium webpage.
Lindsay Gill, Ph.D., our Foundation’s Research Manager attended the Colloquium virtually and stated, “The n-Lorem Foundation is working diligently to provide personalized treatments to patients who are overlooked by large pharmaceutical companies due to the small number of individuals with their particular gene mutation or disease. It is wonderful that a segment of the APBD population may be able to benefit from their groundbreaking work.”