Abstract：
Vamorolone (AGAMREE®) is an oral, selective, dissociative corticosteroid developed by ReveraGen BioPharma and Santhera Pharmaceuticals for the treatment of patients with muscular dystrophy. Vamorolone was approved in October 2023 for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older in the USA and received a positive opinion in the EU in October 2023 for the treatment of DMD in patients 4 years of age and older. This article summarizes the milestones in the development of vamorolone leading to this first approval for DMD.
摘要：
Vamoolone(AGAMREE®)是一种口服，选择性,ReveraGenBioPharma和SantheraPharmaceuticals开发的分离性皮质类固醇，用于治疗肌营养不良患者。Vamoolone于2023年10月被批准用于治疗美国2岁及以上患者的Duchenne肌营养不良症（DMD），并于2023年10月在欧盟获得积极意见，用于治疗4岁及以上患者的DMD。本文总结了vamoolone开发过程中的里程碑，从而首次获得DMD的批准。
