HOME HELP FEEDBACK SUBSCRIPTIONS ARCHIVE SEARCH TABLE OF CONTENTS		QUICK SEARCH:   [advanced] Author: Keyword(s): Year:  Vol:  Page:

This Article Figures Only Full Text Full Text (PDF) Correspondence: Submit a response Alert me when this article is cited Alert me when Correspondence are posted Alert me if a correction is posted Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Citing Articles Citing Articles via HighWire Citing Articles via Google Scholar Google Scholar Articles by Hagemans, M.L.C. Articles by Van der Ploeg, A. T. Search for Related Content PubMed PubMed Citation Articles by Hagemans, M.L.C. Articles by Van der Ploeg, A. T. Related Collections Muscle disease Prognosis

Course of disability and respiratory function in untreated late-onset Pompe disease

From the Department of Metabolic Diseases and Genetics (M.L.C.H., A.T.V.d.P.), Erasmus MC-Sophia, Rotterdam; and the Department of Epidemiology and Biostatistics (W.J.C.H.), Department of Neurology (P.A.V.D.), and Department of Clinical Genetics (M.L.C.H., A.J.J.R.), Erasmus MC, Rotterdam, The Netherlands.

Address correspondence and reprint requests to A.T. van der Ploeg, MD, PhD, Erasmus MC-Sophia, Sp 2435, Dr. Molewaterplein 60, 3015 GJ Rotterdam, The Netherlands; e-mail:a.vanderploeg{at}erasmusmc.nl

Fifty-two untreated patients with late-onset Pompe disease completed questionnaires about their clinical condition and level of handicap at baseline and at 1-year (n = 41) and 2-year follow-ups (n = 40). During this period, declines in functional activities, respiratory function, handicap, and survival were recorded on a group level. This study illustrates the progressiveness of late-onset Pompe disease and indicates the need for close clinical follow-up of both children and adults with this disorder.

Disclosure: This study was a joint initiative of the International Pompe Association (IPA) and Erasmus MC and was financed in part by IPA and Genzyme Corp., Boston, MA. As of August 2004, Drs. Van der Ploeg and Reuser provide consulting services for Genzyme under an agreement between Genzyme and Erasmus MC.

Received August 1, 2005. Accepted in final form November 14, 2005.

This article has been cited by other articles:

				B. Katirji, V. Kesner, R. B. Hejal, and A. Alshekhlee Teaching NeuroImage: Axial muscle atrophy in adult-onset Pompe disease Neurology, March 4, 2008; 70(10): e36 - e36. [Full Text] [PDF]