Tailored antisense oligonucleotides for ultrarare CNS diseases: an experience-based best practice framework for individual patient evaluation - Schüle-Freyer, Rebecca; Graessner, Holm; Aartsma-Rus, Annemieke; van Roon-Mom, Willeke M. C.; Synofzik, Matthis (Molecular therapy. Nucleic Acids, 2025)
Online verfügbar: 1. Juli 2025, Artikelversion: 18. Juli 2025