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264th ENMC International Workshop: Multi-system involvement in spinal muscular atrophy Hoofddorp, the Netherlands, November 19th – 21st 2021

Nora Tula Detering, Alberto Zambon,Niko Hensel,Rashmi Kothary,Kathryn Swoboda,Thomas H. Gillingwater,Giovanni Baranello

Neuromuscular Disorders（2022）

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Abstract
•SMA displays a multi-systemic involvement across different tissues and organs.•Non-neuronal manifestations may contribute to SMA pathology and symptoms.•Unknown multi-system mechanisms are under investigation in preclinical studies.•Novel phenotypes in treated patients warrant refined clinical monitoring and care.•Close interaction between patients, researchers and clinicians needs to be prioritized.
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Key words
spinal muscular atrophy hoofddorp,multi-system
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