Development of a disease severity scoring system for patients with Pompe disease by Giannini E, Berger K, Ploeg A, Case L, Dandrea C, Kishnani P, Marsden D

The use of non-depleting anti-CD4 monoclonal antibody for immune tolerance induction in Pompe disease by Banugaria, S, Patel, T, Prater, SN, Das, S, Boyette, K, Ringler, D, de Fougerolles, A, Koeberl, D, Bali, D, Sun, B, Waldmann, H, Kishnani, P