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

Autor: Hagemans ML; Department of Metabolic Diseases and Genetics, Erasmus MC-Sophia, Rotterdam, The Netherlands., Hop WJ, Van Doorn PA, Reuser AJ, Van der Ploeg AT
Jazyk: angličtina
Zdroj: Neurology [Neurology] 2006 Feb 28; Vol. 66 (4), pp. 581-3.
DOI: 10.1212/01.wnl.0000198776.53007.2c
Abstrakt: 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.
Databáze: MEDLINE