Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes.

Sally Dunaway Young,Amy Pasternak,Tina Duong,Katlyn E McGrattan, Sarah Stranberg, Elizabeth Maczek,Courtney Dias,Whitney Tang,Dana Parker,Alexis Levine, Alyssa Rohan,Connie Wolford,William Martens,Michael P McDermott,Basil T Darras,John W Day

Journal of neuromuscular diseases（2023）

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摘要

Weaker individuals with SMA experience bulbar problems including difficulties with voice and swallowing. Further refinement and assessment of functional bulbar scales will help determine their relevance and responsiveness to changes in SMA. Additional study is needed to quantify bulbar changes caused by SMA and their response to disease-modifying treatments.

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关键词

Spinal muscular atrophy,communication,deglutition,dysphagia,patient-reported outcome measures,speech,swallow,voice

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