A scalable and equitable framework for target and patient prioritisation in rare disease antisense therapeutics
Ella F. Whittle, Kylie Montgomery, Carme Camps, Nour M. Elkhateeb, Catherine Ryan, Sara Aguti, Thales A. C. de Guimarães, Usha Kini, Helen S. Stewart, Andrew G. L. Douglas, Louise C. Wilson, Harry G. Leitch, David S. Lynch, Robert A. Robinson, Michel Michaelides, , Mina Ryten
The paper presents a framework for prioritizing targets and patients for antisense oligonucleotide therapies in rare diseases, focusing on equitable and systematic decision-making.
medRxiv · Mar 26, 2026 · PreprintGenetic & Genomic Medicine