Article

Prominent Respiratory Involvement Featured in High-Risk Screening for Late-Onset Pompe Disease in China

2021-11-29

Abstract excerpt

<title>Abstract</title> <p><bold>Backgroud</bold>: Pompe disease is a rare metabolic disorder with available enzymatic replacement therapy. Contrasting with the classic infantile form, the others subtypes have a heterogeneous presentation that makes an early and accurate diagnosis difficulty. A multicenter observational study was aimed to assess the prevalence of late-onset Pompe disease (LOPD) in high-risk popul...

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Literature Corpus work: c9cacb18-5c14-5b87-9aa5-d359598cf9b1
DOI: 10.21203/rs.3.rs-1104428/v1

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