New motor outcome function measures in evaluation of Late-Onset Pompe disease before and after enzyme replacement therapy
Articolo
Data di Pubblicazione:
2012
Citazione:
New motor outcome function measures in evaluation of Late-Onset Pompe disease before and after enzyme replacement therapy / C. Angelini, C. Semplicini, S. Ravaglia, M. Moggio, G. Comi, O. Musumeci, E. Pegoraro, P. Tonin, M. Filosto, S. Servidei, L. Morandi, G. Crescimanno, G. Marrosu, G. Siciliano, T. Mongini, A. Toscano. - In: MUSCLE & NERVE. - ISSN 0148-639X. - 45:6(2012 Jun), pp. 831-834.
Abstract:
Introduction: The clinical course of late-onset Pompe disease is heterogeneous, and new clinical outcome measures are needed to evaluate enzyme replacement therapy (ERT). Methods: We correlated the 6-Minute Walk Test (6MWT), Walton and Gardner-Medwin (WGM) score, and GSGC (Gait, Stairs, Gower, Chair) scores in 40 patients. Results: At baseline, the GSGC score correlated with both WGM (P < 0.001, n = 33) and 6MWT (P < 0.001, n = 26). After 1 year of ERT, we observed a significant change in gait, stairs and chair performance on the GSGC scale. The 6MWT significantly increased from 319 to 371 meters in 32 patients, and the WGM score was reduced. Conclusions: GSGC is a group of functional tests that requires only a few minutes to perform, therefore, this score might be a good indicator to be used in future studies.
Tipologia IRIS:
01 - Articolo su periodico
Keywords:
enzyme replacement therapy; glycogen storage type II; outcome measures; protocol; trial
Elenco autori:
C. Angelini, C. Semplicini, S. Ravaglia, M. Moggio, G. Comi, O. Musumeci, E. Pegoraro, P. Tonin, M. Filosto, S. Servidei, L. Morandi, G. Crescimanno, G. Marrosu, G. Siciliano, T. Mongini, A. Toscano
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