Disease severity in children and adults with Pompe disease related to age and disease duration.

Hagemans, M L C; Winkel, L P F; Hop, W C J; Reuser, A J J; Van Doorn, P A; Van der Ploeg, A T · Neurology · 2005

cross_sectional · Level IV

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Abstract

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.

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