Dose selection for intracerebroventricular cerliponase alfa in children with CLN2 disease, translation from animal to human in a rare genetic disease

por Hammon, Kevin, de Hart, Greg, Vuillemenot, Brian R., Kennedy, Derek, Musson, Don, O’Neill, Charles A., Katz, Martin L., Henshaw, Joshua W.
Publicado 2021

Clinical Pharmacokinetics and Pharmacodynamics of Cerliponase Alfa, Enzyme Replacement Therapy for CLN2 Disease by Intracerebroventricular Administration

por Kim, Aryun, Grover, Anita, Hammon, Kevin, de Hart, Greg, Slasor, Peter, Cherukuri, Anu, Ajayi, Temitayo, Jacoby, David, Schulz, Angela, Specchio, Nicola, de Los Reyes, Emily, Gissen, Paul, Henshaw, Joshua W.
Publicado 2020

Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer

por Sandza, Krystal, Clark, Annie, Koziol, Elli, Akeefe, Hassibullah, Yang, Fan, Holcomb, Jennifer, Patton, Kathryn, Hammon, Kevin, Mitchell, Nina, Wong, Wing Y., Zoog, Stephen J., Kim, Benjamin, Henshaw, Joshua, Vettermann, Christian
Publicado 2021