Acceleron新药ACE-536获批为罕见病药物
发布时间：[2013/4/1] 访问人数：[1557]
--Acceleron公司和Celgene公司联合研发的ACE-536获准成为治疗贝塔型地中海贫血和骨髓增生异常综合征这两种罕见血液病的罕见病药物。该种药物是作为一种修饰过的二型激活素受体结合蛋白作用于TGF-β来达到治疗效果的。
详细英文报道：
Cambridge, MA-based Acceleron has snagged two orphan indications for ACE-536, an experimental therapy for two rare blood disorders: beta-thalassemia and myelodysplastic syndromes. Acceleron launched its mid-stage study of 536 for beta-thalassemia just days ago. ACE-536, partnered with Celgene ($CELG), is a "modified type II activin receptor fusion protein that acts as a ligand trap for members in the TGF-β superfamily involved in erythropoiesis."