Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study

by van Gelder, C. M., Poelman, E., Plug, I., Hoogeveen-Westerveld, M., van der Beek, N. A. M. E., Reuser, A. J. J., van der Ploeg, A. T.
Published 2016