The clinical course of late-onset Pompe disease is heterogeneous, and new clinical outcome measures are needed to evaluate enzyme replacement therapy (ERT).
New motor outcome function measures in evaluation of late-onset Pompe disease before and after enzyme replacement therapy / Angelini, C., Semplicini, C., Ravaglia, S., Moggio, M., Comi, G.P., Musumeci, O., Pegoraro, E., Tonin, P., Filosto, M., Servidei, S., Morandi, L., Crescimanno, G., Marrosu, G., Siciliano, G., Mongini, T., Toscano, A., Carubbi, F.. - In: MUSCLE & NERVE. - ISSN 0148-639X. - STAMPA. - 45:6(2012), pp. 831-834. [10.1002/mus.23340]
New motor outcome function measures in evaluation of late-onset Pompe disease before and after enzyme replacement therapy
CARUBBI, Francesca
2012
Abstract
The clinical course of late-onset Pompe disease is heterogeneous, and new clinical outcome measures are needed to evaluate enzyme replacement therapy (ERT).
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2012_Angelini-C._Ariatti-A._Muscle-Nerve.pdf
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