我们的第四季度2024更新提供了三名感兴趣的特工的摘要,并在2024年或2025年提供了预期的批准:vanzacaftor/tezacaftor/tezacaftor/deutivacaftor用于囊性纤维化,Crinecerfont,用于先天性肾上腺肾上腺素增生的crinecerfont,用于cretostostimogene grenadenerepvec carenadenenorepvec bladecvec cancer。还突出了管道中其他精选的药物和生物制剂,包括基因疗法和生物仿制药。将介绍有关阿尔茨海默氏病的最新消息。Other topics this quarter include updates on recent respiratory syncytial virus (RSV) and coronavirus disease (COVID-19) vaccine recommendations from the Centers for Disease Control and Prevention (CDC) and an overview of two Food and Drug Administration (FDA) programs: the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program and the Rare Disease Innovation Hub.
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