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。

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）。

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：脊髓性肌萎缩症，肌肉变弱但大脑仍然强壮。