Development of a disease severity scoring system for patients with Pompe disease

por Giannini, E, Berger, K, Ploeg, A van der, Case, L, Dandrea, C, Kishnani, P, Marsden, D
Publicado 2008

The Pompe Registry: tracking Pompe disease symptoms in a broad patient population

por Byrne, B, Kishnani, PS, Case, L, Merlini, L, Müeller-Felber, W, Ploeg, A Van der, Marsden, D, Prasad, S
Publicado 2008