Over time, SMA can make it impossible for the child to move, swallow or breathe.
随着时间的推移,脊萎症会使儿童无法移动、吞咽或呼吸。
The one other medicine for SMA approved in the U.S. is a drug called Spinraza.
美国批准的另一种治疗脊萎症的药物叫做斯宾拉沙(Spinraza)。
In patient testing, babies with the most severe form of SMA who got Zolgensma within 6 months of birth had limited muscle problems.
在病人测试中,患有最严重的脊萎症婴儿在出生后6个月内获得了佐尔根斯玛的治疗,他们现在的肉问题不多。
My condition is known as SMA: spinal muscular atrophy, where the muscles are getting weak but the brain remains strong.
我的病情被称为 SMA:脊萎症,肉变弱但大脑仍然强壮。
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.
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萎症的儿童。
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.
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,从而修复脊萎症的遗传原因。
But now all of a sudden, you're like a Luddite with the textile looms that you just want to sma — what's the problem there, seriously?
但现在突然间, 你就像一个拿着纺织机的勒德分子, 你只想抓住它——这到底是怎么回事,真的吗?
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