Assessment of motor function and nutritional status in children with spinal muscular atrophy treated with nusinersen after loading period in western China Hua Yang ( yanghua202203@163.com ) Sichuan University ...
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...Assessment of motor function and nutritional status in children with spinal muscular atrophy treated nusinersen after loading period western china hua yang yanghua com sichuan university west second hospital department pediatrics qiuji tao dan li xi an jiaotong aliated jie qianyun cai jing gan shaoping huang rong luo research article keywords measure posted date september nd doi https org rs v license this work is licensed under a creative commons attribution international read full page abstract introduction the treatment sma includes disease modifying drugs such as our aim was to summarize experience regarding ecacy safety methods retrospective study data from all patients genetically conrmed diagnosis before years age who were collected initiation months we assessed using standardized scales side effects results aged enrolled had copies survival neuron smn gene type improved have disorders are most malnourished no serious adverse observed conclusion included pediatric especially pro...