INVESTIGATIONAL DRUG ELX-02 MEDIATES CFTR NONSENSE MUTATION READ-THROUGH TO INCREASE CFTR MRNA, CFTR PROTEIN TRANSLATION AND CFTR FUNCTION

Goddeeris, M; Landskroner-Eiger, S; Rot, C; Mullenders, J; Leubitz, A; Banks, K; Williams, G

PEDIATRIC PULMONOLOGY, 2019; 54 (): S212