JOURNAL ARTICLE

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

M L C Hagemans, L P F Winkel, W C J Hop, A J J Reuser, P A Van Doorn, A T Van der Ploeg
Neurology 2005 June 28, 64 (12): 2139-41
15985590
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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