And the ALS ice bucket challenge has now reached presidential levels.

现在肌萎缩化症的冰桶挑战活动已经达了总统级别。

Babies with severe disease don't usually live past their third birthday.

患有严重髓性肌萎缩症的婴儿通常活不过三岁。

She helped care for a brother who had amyotrophic lateral sclerosis (ALS) in his 50s.

她帮助照顾了一位在50多岁患上肌萎缩性化症的兄弟。

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髓性肌萎缩症的药物叫做斯宾拉沙（Spinraza）。

The treatment is for spinal muscular atrophy, a rare inherited disease that affects infants.

该治疗方法适用于髓性肌萎缩症，这是一种影响婴儿的罕见遗传性疾病。

Eight years earlier, Jack had died of amyotrophic lateral sclerosis (ALS), a rare and devastating neurological disease.

八年前，杰克死于肌萎缩性髓化症(ALS)。

Up until now, treatments for spinal muscular atrophy were given after birth.

前为止，治疗髓性肌萎缩症的药物都是在婴儿出生后才服用。

When he was 23, he was diagnosed with ALS, a real disease of the nerves.

当他23岁时，他被诊断出患有肌萎缩化症，一种真正的神经疾病。

It's about as rare as ALS; there are around 7,000 new cases each year in the United States.

它和肌萎缩化症一样罕见；美国每年大约有7000例新病例。

Without the support of dystrophin in place, the sarcolemma essentially wilts and becomes unstable.

没有抗肌萎缩蛋白的支撑，肌膜塌缩变得不稳定。

Over time, SMA can make it impossible for the child to move, swallow or breathe.

随着时间的推移，髓性肌萎缩症会使儿童无法移动、吞咽或呼吸。

This new evidence suggests it might also aid diseases of the nervous system like spinal muscle atrophy, or SMA.

这一新的证据表明，它也可能有助于神经系统疾病，如髓肌萎缩症（SMA）。

She passed away over a decade ago from ALS, six months before I ever released my first YouTube video.

十多年前，在我发布第一支油管视频的 6 个月前，她因为肌萎缩化去世了。

The Food and Drug Administration approved a gene therapy developed by Novartis for treating spinal muscular atrophy in children.

美国食品和药物管理局批准了诺华(Novartis)开发的一种基因疗法，用于治疗儿童髓性肌萎缩症（SMA）。

And his company's A.L.S. Wonder drug is killing people.

他公司治疗肌萎缩性化的灵药吃死人了。

Okay, so as a quick recap: the protein dystrophin is super important for stabilizing the muscle cell membrane.

抗肌萎缩蛋白对肌纤维稳定性的维持极其重要。

But in some rare cases, like Hawking’s, ALS starts earlier in life.

但在一些罕见的情况下，比如霍金，肌萎缩化在生命早期就开始了。

Hawking’s case was also a medical marvel because of how long he lived with ALS.

霍金的病例也是医学上的奇迹，因为他患有肌萎缩化的时间如此之长。

He was the best-known theoretical physicist of his time despite being afflicted with ALS which left him debilitated for decades.

尽管几十年来受肌萎缩化（ALS）的折磨，身体十分虚弱，他仍曾是当下最著名的理论物理学家。

How long have you known Quentin had A.L.S.?

你知道奎汀有肌萎缩化症多久了？