Genetic variation in aryl N-acetyltransferase results in significant differences in the pharmacokinetic and safety profiles of amifampridine (3,4-diaminopyridine) phosphate

por Haroldsen, Peter E, Garovoy, Marvin R, Musson, Donald G, Zhou, Huiyu, Tsuruda, Laurie, Hanson, Boyd, O’Neill, Charles A
Publicado 2015

Formulation and PEGylation optimization of the therapeutic PEGylated phenylalanine ammonia lyase for the treatment of phenylketonuria

por Bell, Sean M., Wendt, Dan J., Zhang, Yanhong, Taylor, Timothy W., Long, Shinong, Tsuruda, Laurie, Zhao, Bin, Laipis, Phillip, Fitzpatrick, Paul A.
Publicado 2017

Maternal phenylketonuria syndrome: studies in mice suggest a potential approach to a continuing problem

por Zeile, William L, McCune, Helen C, Musson, Donald G, O'Donnell, Brian, O'Neill, Charles A, Tsuruda, Laurie S, Zori, Roberto T, Laipis, Philip J
Publicado 2018

The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy

por Long, Brian R., Sandza, Krystal, Holcomb, Jennifer, Crockett, Lucy, Hayes, Gregory M., Arens, Jeremy, Fonck, Carlos, Tsuruda, Laurie S., Schweighardt, Becky, O’Neill, Charles A., Zoog, Stephen, Vettermann, Christian
Publicado 2019

Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice

por Bunting, Stuart, Zhang, Lening, Xie, Lin, Bullens, Sherry, Mahimkar, Rajeev, Fong, Sylvia, Sandza, Krystal, Harmon, Danielle, Yates, Bridget, Handyside, Britta, Sihn, Choong-Ryoul, Galicia, Nicole, Tsuruda, Laurie, O’Neill, Charles A., Bagri, Anil, Colosi, Peter, Long, Shinong, Vehar, Gordon, Carter, Barrie
Publicado 2018