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PTC生物制药公司近日宣布,该公司最新研制的苯基硫脲124(PTC124)药物已获得欧盟医药局(EMEA)孤儿药品委员会(COMP)批准用于杜兴肌营养不良肌肉萎缩症(DMD)和囊性纤维化疾病(CF)治疗的孤儿药开发地位。该公司专门从事靶向基因转录后控制机制的小分子药物设计、研发和商品化生产。
PTC Biopharmaceutical recently announced that its newly developed phenylthiourea 124 (PTC124) drug has been approved by the European Medicines Agency’s (EMEA) Orphan Drug Commission (COMP) for Duchenne muscular dystrophy (DMD) and Cyclofibrosis (CF) treatment of orphan drug development status. The company specializes in the design, development and commercialization of small molecule drugs that target post-transcriptional control mechanisms.