A prospective study of the Erasmus MC University Medical Center (Rotterdam, the Netherlands) shows that Enzyme Replacement Therapy (ERT) has "a positive impact on survival of adult patients with Pompe disease".
Based on an international observational study that provided access to data from 283 adult patients with Pompe disease the authors state that "a patient on ERT has a 59% smaller chance of dying than someone not on ERT".
The study was a joint initiative of the IPA and the Erasmus MC: the International Pompe Association (IPA)/Erasmus MC Pompe Survey, an ongoing international observational follow-up study on the clinical course of patients with Pompe disease, has continually enrolled patients since May 2002. Patients were recruited through patient organizations affiliated with the IPA from Australia, Canada, Germany, the Netherlands, the United Kingdom, the United States, and a small number of patients from other countries.
Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study
Deniz Güngör, Michelle E Kruijshaar, Iris Plug, Ralph B D'Agostino, Marloes LC Hagemans, Pieter A van Doorn, Arnold JJ Reuser and Ans T van der Ploeg
Orphanet Journal of Rare Diseases 2013, 8:49 (doi:10.1186/1750-1172-8-49)
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