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

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

Hagemans ML; Hop WJ; Van Doorn PA; Reuser AJ; Van der Ploeg AT

Neurology

2006

Child; Female; Humans; Male; Adult; Questionnaires; Follow-Up Studies; Aged; Middle Aged; Disease Progression; Disabled Persons; Time Factors; adolescent; Preschool; 80 and over; Q3 Literature Search; Age of Onset; Respiratory Function Tests; Glycogen Storage Disease Type II/physiopathology

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.

2006

Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).

Journal Article

Backlog