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

该治疗方法适用于脊髓性肌萎缩症，这是一种影响婴儿的罕见遗传性疾病。

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脊髓性肌萎缩症的药物叫做斯宾拉沙（Spinraza）。

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

患有严重脊髓性肌萎缩症的婴儿通常活不过三岁。

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到达了总统级别。

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。

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

八年，死于肌萎缩性脊髓化症(ALS)。

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。

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

十多年，在我发布第一支油管视频的 6 个，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，用于治疗儿童脊髓性肌萎缩症（SMA）。

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，如脊髓肌萎缩症（SMA）。

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

到目为止，治疗脊髓性肌萎缩症的药物都是在婴儿出生后才服用。

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

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。

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。

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。

The amyotrophic lateral sclerosis that can find him to a wheelchair.

肌萎缩化症让他坐上了轮椅。

And we've done so many things for the Muscular Dystrophy Association over the years.

多年来，我们为肌萎缩化症协会做了许多事情。

ALS, its official name is amyotrophic lateral sclerosis.

ALS的正式名称是肌萎缩化症。

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

你知道奎汀有肌萎缩化症多久了？

Even today, you get A.L.S., within three to five years, you die.

就算今时今日 患上肌萎缩化症，不出三五年也必死无疑。