Preclinical Evaluation of ADVM-022, a Novel Gene Therapy Approach to Treating Wet Age-Related Macular Degeneration

por Grishanin, Ruslan, Vuillemenot, Brian, Sharma, Pallavi, Keravala, Annahita, Greengard, Judith, Gelfman, Claire, Blumenkrantz, Mark, Lawrence, Matthew, Hu, Wenzheng, Kiss, Szilárd, Gasmi, Mehdi
Publicado 2019

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

Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease)

por Katz, Martin L, Coates, Joan R, Sibigtroth, Christine M, Taylor, Jacob D, Carpentier, Melissa, Young, Whitney M, Wininger, Fred A, Kennedy, Derek, Vuillemenot, Brian R, O'Neill, Charles A
Publicado 2014

Preclinical pharmacokinetics and pharmacodynamics of DCLL9718A: An antibody-drug conjugate for the treatment of acute myeloid leukemia

por Leipold, Douglas D., Figueroa, Isabel, Masih, Shabkhaiz, Latifi, Brandon, Yip, Victor, Shen, Ben-Quan, Dere, Randall C., Carrasco-Triguero, Montserrat, Lee, M. Violet, Saad, Ola M., Liu, Luna, He, Jintang, Su, Dian, Xu, Keyang, Vuillemenot, Brian R., Laing, Steven T., Schutten, Melissa, Kozak, Katherine R., Zheng, Bing, Polson, Andrew G., Kamath, Amrita V.
Publicado 2018

Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB

por Ellinwood, N. Matthew, Valentine, Bethann N., Hess, Andrew S., Jens, Jackie K., Snella, Elizabeth M., Jamil, Maryam, Hostetter, Shannon J., Jeffery, Nicholas D., Smith, Jodi D., Millman, Suzanne T., Parsons, Rebecca L., Butt, Mark T., Chandra, Sundeep, Egeland, Martin T., Assis, Ana B., Nelvagal, Hemanth R., Cooper, Jonathan D., Nestrasil, Igor, Mueller, Bryon A., Labounek, Rene, Paulson, Amy, Prill, Heather, Liu, Xiao Ying, Zhou, Huiyu, Lawrence, Roger, Crawford, Brett E., Grover, Anita, Cherala, Ganesh, Melton, Andrew C., Cherukuri, Anu, Vuillemenot, Brian R., Wait, Jill C.M., O’Neill, Charles A., Pinkstaff, Jason, Kovalchin, Joseph, Zanelli, Eric, McCullagh, Emma
Publicado 2022