A role for plasma cell targeting agents in immune tolerance induction in autoimmune disease and antibody responses to therapeutic proteins by Rosenberg, A.S, Pariser, A.R, Diamond, B, Yao, L, Turka, L.A, Lacana, E, Kishnani, P.S

Get full text

Emerging therapies for glycogen storage disease type I by Koeberl, D.D, Kishnani, P.S, Bali, D, Chen, Y-T

Get full text

S.P.22 Coloring muscle weakness “CMW” a new tool in neuromuscular diseases by Corderi, J.R, Dubrovsky, A, Kishnani, P.S, Case, L, Javan, R, Horvath, J

Get full text

P.17.5 Analysis of motor assessments administered at treatment initiation for patients in the pompe registry by van der Ploeg, A, Angelini, C, Kishnani, P.S, Amartino, H, Müller-Felber, W, Kruijshaar, M.E, Devecseri, Z, Miller, T, Wilson, A, Case, L.E

Get full text

M.P.1.08 Diagnosis of Pompe disease: Timing and methods used as reported to the Pompe registry by Kishnani, P.S, Miller, T, Prasad, S

Get full text

Important role of abnormal glycogen structure in the development of liver cirrhosis and progression to hepatocellular carcinoma in patients with glycogen storage disease type-III by Kishnani, P.S, Chen, Y.T

Get full text

Immunomodulatory, liver depot gene therapy for Pompe disease by Bond, J.E., Kishnani, P.S., Koeberl, D.D.

Get full text

G.P.16.01. The Pompe registry: Tracking Pompe disease symptoms in a broad patient population by Byrne, B, Kishnani, P.S, Case, L, Merlini, L, Müeller-Felber, W, der Ploeg, A. Van

Get full text

G.P.11.01 Alglucosidase alfa in infants and children with Pompe disease by Wraith, E, Byrne, B, Hwu, W.L, Leslie, N, Mandel, H, Nicolino, M, Kishnani, P.S

Get full text

G.P.8.01 Diagnosis of Pompe disease using a rapid dried blood spot assayin patients with muscle weakness by Goldstein, J.L, Young, S.P, Changela, M, Dickerson, G.H, Zhang, H, Dai, J, Peterson, D, Millington, D.S, Prasad, S, Kishnani, P.S, Bali, D

Get full text

HLA- and genotype-based risk assessment model to identify infantile onset pompe disease patients at high-risk of developing significant anti-drug antibodies (ADA) by De Groot, A.S., Kazi, Z.B., Martin, R.F., Terry, F.E., Desai, A.K., Martin, W.D., Kishnani, P.S.

Get full text

A randomized trial comparing the efficacy and safety of imiglucerase (Cerezyme) infusions every 4 weeks versus every 2 weeks in the maintenance therapy of adult patients with Gauch... by Kishnani, P.S., DiRocco, M., Kaplan, P., Mehta, A., Pastores, G.M., Smith, S.E., Puga, A.C., Lemay, R.M., Weinreb, N.J.

Get full text