The influence of a polymorphism in the gene encoding angiotensin converting enzyme (ACE) on treatment outcomes in late-onset Pompe patients receiving alglucosidase alfa

por Baek, Rena C., Palmer, Rachel, Pomponio, Robert J., Lu, Yuefeng, Ma, Xiwen, McVie-Wylie, Alison J.
Publicado 2016

Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis

por Boye, Sanford L., O’Riordan, Catherine, Morris, James, Lukason, Michael, Compton, David, Baek, Rena, Elmore, Dana M., Peterson, James.J., Fajardo, Diego, McCullough, K. Tyler, Scaria, Abraham, McVie-Wylie, Alison, Boye, Shannon E.
Publicado 2022

An immune tolerance approach using transient low-dose methotrexate in the ERT-naïve setting of patients treated with a therapeutic protein: experience in infantile-onset Pompe dise...

por Kazi, Zoheb B., Desai, Ankit K., Troxler, R. Bradley, Kronn, David, Packman, Seymour, Sabbadini, Marta, Rizzo, William B., Scherer, Katalin, Abdul-Rahman, Omar, Tanpaiboon, Pranoot, Nampoothiri, Sheela, Gupta, Neerja, Feigenbaum, Annette, Niyazov, Dmitriy M., Langston, Sherry, Segel, Reeval, McVie-Wylie, Alison, Sung, Crystal, Joseph, Alexandra M., Richards, Susan, Kishnani, Priya S.
Publicado 2018