If you have made a diagnosis of spinal muscular atrophy (SMA) type 1 (also known as Werdnig–Hoffman disease) in a child, then you have vivid memories of informing desperate parents that there is nothing you can do for their child. Two innovative therapies for SMA may now bring some hope — but what do they mean for patients and their families? SMA, which is one of the most common inheritable neuromuscular diseases, is a degenerative motor neuron disorder that leads to muscle atrophy and respiratory failure. Patients with the most severe form rarely survive beyond 2 years of age. The . . .

中文翻译：

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脊柱肌肉萎缩的两种创新疗法的困境

如果您已诊断出一名儿童患有1型脊髓性肌萎缩症（SMA）（也称为Werdnig-Hoffman病），那么您会生动地告诉绝望的父母他们无法为他们的孩子做任何事情。SMA的两种创新疗法现在可能会带来一些希望-但是它们对患者及其家人意味着什么？SMA是最常见的遗传性神经肌肉疾病之一，是一种退化性运动神经元疾病，可导致肌肉萎缩和呼吸衰竭。具有最严重形式的患者很少能存活到2岁以上。这 。。。