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Clinical features of late-onset Pompe disease: a prospective cohort study.
Muscle Nerve 2008; 38(4):1236-45MN

Abstract

The objective of this 12-month study was to describe the clinical features of late-onset Pompe disease and identify appropriate outcome measures for use in clinical trials. Assessments included quantitative muscle testing (QMT), functional activities (FAA), 6-min walk test (6MWT), and pulmonary function testing (PFT). Percent predicted values indicated quantifiable upper and lower extremity weakness, impaired walking ability, and respiratory muscle weakness. Significant declines in arm and leg strength and pulmonary function were observed during the study period. The outcome measures were demonstrated to be safe and reliable. Symptom duration was identified as the best predictor of the extent of skeletal and respiratory muscle weakness.

Authors+Show Affiliations

Universitair Medisch Centrum Utrecht HP G03.228, Heidelberglaan 100, 3584 CX Utrecht, Netherlands. J.Wokke@umcutrecht.nlNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Multicenter Study
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

18816591

Citation

Wokke, John H J., et al. "Clinical Features of Late-onset Pompe Disease: a Prospective Cohort Study." Muscle & Nerve, vol. 38, no. 4, 2008, pp. 1236-45.
Wokke JH, Escolar DM, Pestronk A, et al. Clinical features of late-onset Pompe disease: a prospective cohort study. Muscle Nerve. 2008;38(4):1236-45.
Wokke, J. H., Escolar, D. M., Pestronk, A., Jaffe, K. M., Carter, G. T., van den Berg, L. H., ... Laforet, P. (2008). Clinical features of late-onset Pompe disease: a prospective cohort study. Muscle & Nerve, 38(4), pp. 1236-45. doi:10.1002/mus.21025.
Wokke JH, et al. Clinical Features of Late-onset Pompe Disease: a Prospective Cohort Study. Muscle Nerve. 2008;38(4):1236-45. PubMed PMID: 18816591.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Clinical features of late-onset Pompe disease: a prospective cohort study. AU - Wokke,John H J, AU - Escolar,Diana M, AU - Pestronk,Alan, AU - Jaffe,Kenneth M, AU - Carter,Gregory T, AU - van den Berg,Leonard H, AU - Florence,Julaine M, AU - Mayhew,Jill, AU - Skrinar,Alison, AU - Corzo,Deyanira, AU - Laforet,Pascal, PY - 2008/9/26/pubmed PY - 2008/12/17/medline PY - 2008/9/26/entrez SP - 1236 EP - 45 JF - Muscle & nerve JO - Muscle Nerve VL - 38 IS - 4 N2 - The objective of this 12-month study was to describe the clinical features of late-onset Pompe disease and identify appropriate outcome measures for use in clinical trials. Assessments included quantitative muscle testing (QMT), functional activities (FAA), 6-min walk test (6MWT), and pulmonary function testing (PFT). Percent predicted values indicated quantifiable upper and lower extremity weakness, impaired walking ability, and respiratory muscle weakness. Significant declines in arm and leg strength and pulmonary function were observed during the study period. The outcome measures were demonstrated to be safe and reliable. Symptom duration was identified as the best predictor of the extent of skeletal and respiratory muscle weakness. SN - 0148-639X UR - https://www.unboundmedicine.com/medline/citation/18816591/Clinical_features_of_late_onset_Pompe_disease:_a_prospective_cohort_study_ L2 - https://doi.org/10.1002/mus.21025 DB - PRIME DP - Unbound Medicine ER -