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PTC124(Ataluren)
Selective inducer of stop codon read-through
GLPG1837(ABBV-974)
A CFTR potentiator
GlyH-101
A reversible CFTR channel blocker
VX-661
An investigational compound that promotes the maturation of delta F508 CFTR
(R)-BPO-27
(R)-BPO-27是有效的CFTR抑制劑,IC50值為4nM。
PPQ-102
A CFTR channel inhibitor
CFTR corrector 1
An F508del-CFTR corrector and potentiator
CFTRinh-172
A potent CFTR channel blocker
CFTR corrector 2
FDL169 is a CFTR corrector that is designed to fix and restore the function of the defective CFTR protein.
DNA31
DNA31 是囊性纖維化跨膜傳導調節因子 (CFTR) 基因的全球最常見的 31 種突變。DNA31 用于針對囊性纖維化 (CF) 的新生兒篩查方案的第一級遺傳測試。
BPO-27 racemate
BPO-27racemate是有效的CFTR抑制劑,IC50值為8nM。
PTI-428
Nesolicaftor (PTI-428) is a specific cystic fibrosis transmembrane conductance regulator (CFTR) amplifier that increases CFTR expression.
VX-809
A F508del-CFTR corrector
IOWH-032
A modulator of CFTR
GLPG2451
GLPG2451 是一種囊性纖維化跨膜電導調節器 (CFTR) 的增強劑,有效增強低溫挽救的 F508del CFTR,EC50 為 11.1 nM。
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