治疗杜氏肌营养不良的新药——依替来生
发布时间：2019-01-05 15:42
【摘要】：杜氏肌营养不良是由于编码抗肌萎缩蛋白的基因突变引起,抗肌萎缩蛋白可避免肌肉收缩放松过程中的肌肉细胞损伤。依替来生为一种反义寡核苷酸,作用于抗肌萎缩蛋白前体信使RNA,使其在转录过程中跳过外显子51,从而使杜氏肌营养不良患者产生了缩短的但具有一定功能的肌营养不良蛋白。依替来生由Sarepta制药公司开发,于2016年9月获美国食品和药物管理局加速批准,用于治疗杜氏肌营养不良。最常见的不良反应主要包括平衡失调、恶心及接触性皮炎等。
[Abstract]:Duchenne muscular dystrophy is caused by a gene mutation that encodes anti-muscular dystrophy protein which can avoid muscle cell damage during muscle contraction and relaxation. Etidine is an antisense oligodeoxynucleotide acting on the anti-muscular dystrophin precursor messenger RNA, which skips exon 51 during transcription. Thus, the patients with Duchenne muscular dystrophy produce shortened muscular dystrophy protein with certain function. Etidyl, developed by Sarepta Pharmaceuticals, was accelerated by the Food and Drug Administration in September 2016 for the treatment of Duchenne muscular dystrophy. The most common adverse reactions include imbalance, nausea and contact dermatitis.
【作者单位】： 临沂市中心医院药学部;
【分类号】：R971
，
本文编号：2401967
[Abstract]:Duchenne muscular dystrophy is caused by a gene mutation that encodes anti-muscular dystrophy protein which can avoid muscle cell damage during muscle contraction and relaxation. Etidine is an antisense oligodeoxynucleotide acting on the anti-muscular dystrophin precursor messenger RNA, which skips exon 51 during transcription. Thus, the patients with Duchenne muscular dystrophy produce shortened muscular dystrophy protein with certain function. Etidyl, developed by Sarepta Pharmaceuticals, was accelerated by the Food and Drug Administration in September 2016 for the treatment of Duchenne muscular dystrophy. The most common adverse reactions include imbalance, nausea and contact dermatitis.
【作者单位】： 临沂市中心医院药学部;
【分类号】：R971
，
本文编号：2401967
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