Genetic & Genomic MedicinemedRxivPreprint
A scalable and equitable framework for target and patient prioritisation in rare disease antisense therapeutics
Ella F. Whittle, Kylie Montgomery, Carme Camps…·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.