直到Vosoritide的获批,才让ACH治疗迈入一个全新的靶向治疗时代。它是一种C型利钠肽(CNP)类似物,通过结合NPR-B进而降低FGFR3的活性来促进生长。临床结果试验显示:相较于安慰剂,每日皮下注射15 μg/kg的Vosoritide,受试者平均年身高增加了1.57厘米。
"We are quite different people - very much yin and yang - but I think decisions are better made with two brains rather than one as it stops hubris," says Begg, who is London-based.
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