除了小分子抑制剂,核酸技术也开始在ACH治疗中得到应用。Ribomic开发的RBM-007,通过限制FGF2和FGFR3激活变体之间的过度相互作用发挥功能。2期临床结果显示,RBM-007的疗效与Vosoritide相近(1.5厘米/年),甚至有一名受试者年增长5厘米,且该试验中包括2周一次的注射频率,是当前在研药物中最长的给药间隔。
"Something has changed," wrote Dorsey. "We're already seeing that the intelligence tools we’re creating and using, paired with smaller and flatter teams, are enabling a new way of working which fundamentally changes what it means to build and run a company. and that's accelerating rapidly."
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