Subject
Child; Cross-Sectional Studies; Female; Humans; infant; Male; Adult; Questionnaires; Aged; Middle Aged; Disease Progression; Age Factors; Respiration; Severity of Illness Index; Activities of Daily Living; adolescent; Preschool; 80 and over; infant; Q3 Literature Search; Newborn; Age of Onset; Muscle; Artificial/statistics & numerical data; Glycogen Storage Disease Type II/diagnosis/epidemiology/physiopathology; Respiratory Insufficiency/epidemiology/etiology/therapy; Skeletal/physiopathology; Wheelchairs/statistics & numerical data
Description
Information about 255 children and adults with Pompe disease was gathered through a questionnaire. Disease severity was associated with disease duration and not with age; an early manifestation of the disease implied earlier wheelchair or ventilator dependency. The patient group under age 15 included a subgroup with a more severe and rapid course of the disease. They require more intensive follow-up and early intervention, before irreversible damage has occurred.
2005