2022-04-24

Porphyria can be so painful that people take potent1 opioid painkillers2 every day. Severe attacks need hospital treatment.

卟啉病可造成强烈的疼痛，以至于患者每天都要服用强效的类鸦片止痛药。严重发作时甚至需要入院治疗。

Rather than managing the pain, gene3 silencing gets to the root cause of the genetic4 disease and the build-up of toxic5 proteins in the body.

基因沉寂技术不是要控制病痛，而是从这种遗传病的根本病因着手治疗，并对抗有毒蛋白质在体内的蓄积。

The drug does not alter people's DNA6 but mutes parts of it to block that protein production. Trials showed it helped cut attacks by 75%.

该药物不会改变病人的 DNA（脱氧核糖核酸），但会抑制部分 DNA，从而阻止这类蛋白质的产生。试验表明，它帮助减少了75%的病症发作。

The National Institute for Health and Care Excellence7, which advises on drugs in England, said the therapy would improve people's quality of life and was value for money.

为英格兰地区提供药物咨询的国家卫生与保健优化研究所表示，这种治疗方法将改善人们的生活质量，且性价比高。