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Untreated adults with Pompe Disease have a higher mortality than the general population

Author: 
IPA
Category: Archive
Published
June 6, 2011

A new scientific paper on “Survival and associated factors in 268 adults with Pompe Disease prior to treatment with enzyme replacement therapy” was published by Deniz Güngör (Erasmus MC, Rotherdam, The Netherlands) and co-workers on June 1, 2011. The authors come to the conclusion that “untreated adults with Pompe Disease have a higher mortality than the general population and that their levels of disability and handicap/participation are the most important factors associated with mortality.”

Open access to the article: Orphanet Journal of Rare Diseases 2011, 6:34.
Currently, a provisional PDF is available, the full article is expected to be available soon.

The IPA has listed the article on its website under Selected Scientific Papers.

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