
Keywords: موش Mdx; Cystic fibrosis; Cystic fibrosis transmembrane conductance regulator protein; CFTR2; Correctors; Potentiators; Trial end-points; Amil; Amiloride; AMP; Accelerating Medicine Partnership; ANO; anoctamins; AONs; antisense oligonucleotides; ASL; airway surfac