Enhancement of CFTR nonsense mutation readthrough via combined small molecule and ACE-tRNA therapies

Translarna (ataluren) - PTC Therapeutics

SRI-41315 - Southern Research

eragidomide (CC-90009) - BMS

https://jcf-25-s2.elsevierdigitaledition.com/

K. Thrasher 1; N. Ward 1; J. Porter 1; D. Bedwell 2; J. Lueck 3

Sep 9, 2026