Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy

Overview of attention for article published in Orphanet Journal of Rare Diseases, June 2011

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Cost-effectiveness of ERT in adult patients with Pompe disease

On Medicine, 19 Dec 2017

Pompe disease (or glycogenosis type II) is a rare inheritable progressive muscle disease caused by a deficiency of the enzyme…

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