治療杜氏肌營養(yǎng)不良的新藥——依替來生
發(fā)布時間：2019-01-05 15:42
【摘要】：杜氏肌營養(yǎng)不良是由于編碼抗肌萎縮蛋白的基因突變引起,抗肌萎縮蛋白可避免肌肉收縮放松過程中的肌肉細胞損傷。依替來生為一種反義寡核苷酸,作用于抗肌萎縮蛋白前體信使RNA,使其在轉(zhuǎn)錄過程中跳過外顯子51,從而使杜氏肌營養(yǎng)不良患者產(chǎn)生了縮短的但具有一定功能的肌營養(yǎng)不良蛋白。依替來生由Sarepta制藥公司開發(fā),于2016年9月獲美國食品和藥物管理局加速批準,用于治療杜氏肌營養(yǎng)不良。最常見的不良反應主要包括平衡失調(diào)、惡心及接觸性皮炎等。
[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.
【作者單位】： 臨沂市中心醫(yī)院藥學部;
【分類號】：R971
，
本文編號：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.
【作者單位】： 臨沂市中心醫(yī)院藥學部;
【分類號】：R971
，
本文編號：2401967
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