Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials.

Neuromuscular Disorders（2016）

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摘要

•The paper reports for the first time patterns of progression in type 2 and 3 SMA.•Different trajectories can be identified in ambulant and non-ambulant patients.•Age appears to be an important factor in determining trajectories of progression.

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关键词

Spinal muscular atrophy,Hammersmith Functional Motor Scale,Outcome measures

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