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November 22, 2017SHILAP Revista de lepidopterologíaOpen Access

The humanistic burden of Pompe disease: are there still unmet needs? A systematic review

Benedikt Schoser Deborah A. Bilder David Dimmock Digant Gupta Emma S. James Suyash Prasad

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Abstract

Pompe disease has a substantial humanistic burden, with strong inter-relationships among and between humanistic burden parameters and clinical progression.

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Schoser et al. (2017) studied this question.

synapsesocial.com/papers/69d6b4b2c2431583c1ab31c6 https://doi.org/10.1186/s12883-017-0983-2

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Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1Cognitive outcome of patients with classic infantile Pompe disease receiving enzyme therapyCohort Study · 2012 · 75 citations
2Facial‐muscle weakness, speech disorders and dysphagia are common in patients with classic infantile Pompe disease treated with enzyme therapyOriginal Research · 2011 · 94 citations
3Pompe Disease: Early Diagnosis and Early Treatment Make a DifferencePrognostic Study · 2013 · 170 citations
4The Natural Course of Infantile Pompe’s Disease: 20 Original Cases Compared With 133 Cases From the LiteratureOriginal Research · 2003 · 555 citations
5Toward deconstructing the phenotype of late‐onset Pompe diseaseOriginal Research · 2012 · 120 citations

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