VOA慢速英语2019 世界上最昂贵的药物是基因治疗，价值200万美元

World’s Costliest¹ Medicine Is Gene² Treatment Priced at $2 Million

U.S. officials have approved the costliest medicine in history – a $2.1-million gene therapy that treats a rare childhood disease.

美国官员批准了史上最昂贵的药物——一种治疗罕见儿童疾病的基因疗法，价值210万美元。 

Gene therapy is a medical treatment in which healthy genetic³ material is introduced into cells to replace abnormal genes⁴.

基因治疗是一种将健康的遗传物质导入细胞以取代异常基因的医学治疗方法。 

The new gene therapy targets a disease called spinal⁵ muscular atrophy⁶ or SMA, which severely⁷ weakens a child’s muscles.The treatment, called Zolgensma, was developed by Swiss drugmaker Novartis.

这项新基因疗法针对的是一种名为脊髓性肌萎缩症(SMA)的疾病，这种疾病会严重削弱儿童的肌肉。这种名为佐尔根斯玛（Zolgensma）的药物是由瑞士制药公司诺华研发。

Over time, SMA can make it impossible for the child to move, swallow or breathe.The disease affects about 1 in 10,000 births.In 90 percent of cases, the condition causes death or the need for permanent breathing assistance by age two.

随着时间的推移，脊髓性肌萎缩症会使儿童无法移动、吞咽或呼吸。每1万名新生儿中就有一人感染这种疾病。在90%的病例中，这种情况会导致死亡，或者在两岁前需要永久性的呼吸辅助。

SMA is the leading cause of genetic infant death.

脊髓性肌萎缩症是婴儿遗传死亡的主要原因。 

The U.S. Food and Drug Administration or FDA recently approved Zolgensma for use in children who are under the age of two with SMA.The approval covers babies with the deadliest kind of the disease as well as those with forms that develop more slowly over time.

美国食品和药物管理局最近批准可以用佐尔根斯玛用于治疗两岁以下患有脊髓性肌萎缩症的儿童。这项批准覆盖的患者包括患有这种最致命疾病，以及那些随着时间推移发育较慢的婴儿。

Novartis says Zolgensma is designed to repair the genetic cause of SMA by replacing a patient’s abnormal or missing gene to halt progression of the disease.The one-time treatment can be performed in about an hour.

诺华表示，佐尔根斯玛旨在通过替换患者异常或缺失的基因来阻止疾病的进展，从而修复脊髓性肌萎缩症的遗传原因。一次性治疗约1小时即可完成。

Novartis has defended the high cost of the gene therapy.It says the treatment represents a new kind of "transformational medicine" that opens the door to future gene therapies.It also said the $2.1-million therapy was about 50 percent less than other SMA treatments that can be ongoing⁹ for years.

诺华为基因治疗的高成本进行了辩护。它说，这种疗法代表了一种新型的“转化医学”，为未来的基因疗法打开了大门。该公司还表示，210万美元的治疗费用比其他持续数年的脊髓性肌萎缩症治疗费用低50%左右。

Novartis said it will let health insurance companies break up the cost in payments of $425,000 over five years.It also plans to give money back if the treatment does not work.

诺华表示，它将让医疗保险公司在未来5年内分摊42.5万美元的保费。如果治疗无效，该公司还计划退款。

The FDA said the drug’s safety had been tested in completed and ongoing trials involving 36 patients between the ages of two weeks and eight months.

美国食品和药物管理局表示，该药物的安全性已经在36名年龄在两周至八个月之间的患者身上进行了测试。

Most evidence of the treatment’s effectiveness is based on the ongoing trials.In patient testing, babies with the most severe form of SMA who got Zolgensma within 6 months of birth had limited muscle problems.

治疗效果的大多数证据都是基于正在进行的试验。在病人测试中，患有最严重的脊髓性肌萎缩症婴儿在出生后6个月内获得了佐尔根斯玛的治疗，他们现在的肌肉问题不多。

Those who got the treatment very early in life did the best.Babies who were given Zolgensma after six months stopped losing muscle control, but the medicine was not able to correct damage already done.

那些在生命早期接受治疗的人表现最好。服用佐尔根斯玛六个月后的婴儿肌肉不再失去控制，但药物无法治疗已经造成的损伤。

The trial found the treatment "demonstrated significant improvement in their ability to reach developmental motor milestones¹¹."These included head control and the ability to sit without support.

试验发现，这种疗法“显示出他们达到发育性运动里程碑的能力有显著改善。”包括控制头部和在没有支撑的情况下坐着的能力。

The one other medicine for SMA approved in the U.S. is a drug called Spinraza.Instead of a one-time treatment, it must be given every four months.The U.S.-based Biogen, Spinraza’s maker⁸, charges $750,000 for the first year of treatment, followed by $350,000 per year after that.

美国批准的另一种治疗脊髓性肌萎缩症的药物叫做斯宾拉沙（Spinraza）。它不能一次性治愈，必须每四个月治疗一次。斯宾拉沙的制造商、总部位于美国的生物基因公司表示，第一年的治疗费用为75万美元，之后每年收费35万美元。

The independent not-for-profit group Institute for Clinical and Economic Review rates the value of costly¹² new medicines.The group said it considers the price of Zolgensma acceptable because it greatly "transforms the lives of families affected¹³ by this devastating¹⁴ disease."

独立的非营利组织临床与经济评论协会评估了昂贵新药的价值。该组织表示，他们认为佐尔根斯玛的价格是可以接受的，因为它“极大地改变了受这种毁灭性疾病影响的家庭的生活”。

While it is too early to know how long the effectiveness of the treatment will last, some doctors have hope that it could last a lifetime.Dr. Jerry Mendell is a neurologist at Nationwide Children’s Hospital in Columbus, Ohio.He led one of the early patient studies involving Zolgensma.

虽然现在还不知道这种疗法的效果能持续多久，但一些医生希望这种疗法能持续一辈子。杰里·门德尔博士是俄亥俄州哥伦布市全国儿童医院的神经学家。他领导了一项关于佐尔根斯玛治疗的早期病人研究。

When asked whether he believes the drug will continue to have long-lasting effects, Mendell told the Associated Press, "it’s beginning to look that way."

当被问及他是否相信这种药物会继续产生长期效果时，门德尔告诉美联社，“情况正朝着这个方向发展。”

He noted¹⁵ that some of those treated as babies are now 4 or 5 years old with no signs of the disease.

他指出，一些接受婴儿治疗的儿童现在已经四、五岁了，没有任何患病迹象。 

Words in This Story

infant – n.a baby or very young child

transformational – adj.able to produce a big change or improvement in a situation

significant – adj.important, large or great

milestone¹⁰ – n.an important event in the development or history of something

devastating – adj.causing great damage of suffering

1 costliest   

adj.昂贵的( costly的最高级 )；代价高的；引起困难的；造成损失的

参考例句：

• At 81 billion dollars, Katrina is the costliest natural disaster in American history. “卡特里娜”飓风造成了近810亿美圆的损失，是美国历史上最严重的自然灾难之一。 来自互联网
• Senator John Kerry has proposed a tax on the costliest health plans sold by insurance companies. 参议员约翰？克里(JohnKerry)已经提议对保险公司销售的高价值的保险计划征税。 来自互联网

2 gene   

n.遗传因子，基因

参考例句：

• A single gene may have many effects.单一基因可能具有很多种效应。
• The targeting of gene therapy has been paid close attention.其中基因治疗的靶向性是值得密切关注的问题之一。

3 genetic   

adj.遗传的，遗传学的

参考例句：

• It's very difficult to treat genetic diseases.遗传性疾病治疗起来很困难。
• Each daughter cell can receive a full complement of the genetic information.每个子细胞可以收到遗传信息的一个完全补偿物。

4 genes   

n.基因( gene的名词复数 )

参考例句：

• You have good genes from your parents, so you should live a long time. 你从父母那儿获得优良的基因，所以能够活得很长。 来自《简明英汉词典》
• Differences will help to reveal the functions of the genes. 它们间的差异将会帮助我们揭开基因多种功能。 来自英汉非文学 - 生命科学 - 生物技术的世纪

5 spinal   

adj.针的，尖刺的，尖刺状突起的；adj.脊骨的，脊髓的

参考例句：

• After three days in Japan,the spinal column becomes extraordinarily flexible.在日本三天，就已经使脊椎骨变得富有弹性了。
• Your spinal column is made up of 24 movable vertebrae.你的脊柱由24个活动的脊椎骨构成。

6 atrophy   

n./v.萎缩，虚脱，衰退

参考例句：

• Patients exercised their atrophied limbs in the swimming pool.病人们在泳池里锻炼萎缩的四肢。
• Many hoped he would renew the country's atrophied political system.很多人都期望他能使该国萎靡的政治体系振作起来。

7 severely   

adv.严格地；严厉地；非常恶劣地

参考例句：

• He was severely criticized and removed from his post.他受到了严厉的批评并且被撤了职。
• He is severely put down for his careless work.他因工作上的粗心大意而受到了严厉的批评。

8 maker   

n.制造者，制造商

参考例句：

• He is a trouble maker，You must be distant with him.他是个捣蛋鬼，你不要跟他在一起。
• A cabinet maker must be a master craftsman.家具木工必须是技艺高超的手艺人。

9 ongoing   

adj.进行中的，前进的

参考例句：

• The problem is ongoing.这个问题尚未解决。
• The issues raised in the report relate directly to Age Concern's ongoing work in this area.报告中提出的问题与“关心老人”组织在这方面正在做的工作有直接的关系。

10 milestone   

n.里程碑；划时代的事件

参考例句：

• The film proved to be a milestone in the history of cinema.事实证明这部影片是电影史上的一个里程碑。
• I think this is a very important milestone in the relations between our two countries.我认为这是我们两国关系中一个十分重要的里程碑。

11 milestones   

n.重要事件( milestone的名词复数 )；重要阶段；转折点；里程碑

参考例句：

• Several important milestones in foreign policy have been passed by this Congress and they can be chalked up as major accomplishments. 这次代表大会通过了对外政策中几起划时代的事件，并且它们可作为主要成就记录下来。 来自《简明英汉词典》
• Dale: I really envy your milestones over the last few years, Don. 我真的很羡慕你在过去几年中所建立的丰功伟绩。 来自互联网

12 costly   

adj.昂贵的，价值高的，豪华的

参考例句：

• It must be very costly to keep up a house like this.维修这么一幢房子一定很昂贵。
• This dictionary is very useful,only it is a bit costly.这本词典很有用，左不过贵了些。

13 affected   

adj.不自然的，假装的

参考例句：

• She showed an affected interest in our subject.她假装对我们的课题感到兴趣。
• His manners are affected.他的态度不自然。

14 devastating   

adj.毁灭性的，令人震惊的，强有力的

参考例句：

• It is the most devastating storm in 20 years.这是20年来破坏性最大的风暴。
• Affairs do have a devastating effect on marriages.婚外情确实会对婚姻造成毁灭性的影响。

15 noted   

adj.著名的，知名的

参考例句：

• The local hotel is noted for its good table.当地的那家酒店以餐食精美而著称。
• Jim is noted for arriving late for work.吉姆上班迟到出了名。