If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use. ...
治疗囊性纤维化跨膜传导调节因子介导疾病的复方新药Symdeko®由美国Vertex 生物技术制药公司研制,含tezacaftor 100 mg和ivacaftor 150 mg的复方片剂及ivacafto 150 mg单一片剂的共包装。2018年2月12日,美国食品药品管理局(FDA)授予该药突破性治疗药物地位的认定,同日批准上市。该药同时向欧洲药品管理局 (EMA)提出...
placebo-controlled, clinical trials: two parallel-group trials of 12 and 24 week duration and one cross-over design trial of 8 weeks duration. Eligible patients were also able to participate in an open-label extension safety study (up to 96 weeks of SYMDEKO). In the three placebo...