1. Dose selection for intracerebroventricular cerliponase alfa in children with CLN2 disease, translation from animal to human in a rare genetic disease. Issue 5 (2nd June 2021) Authors: Hammon, Kevin; de Hart, Greg; Vuillemenot, Brian R.; Kennedy, Derek; Musson, Don; O'Neill, Charles A.; Katz, Martin L.; Henshaw, Joshua W. Journal: Clinical and translational science Issue: Volume 14:Issue 5(2021) Page Start: 1810 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Enzyme replacement therapy attenuates disease progression in a canine model of late‐infantile neuronal ceroid lipofuscinosis (CLN2 disease). Issue 11 (17th June 2014) Authors: 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. Journal: Journal of neuroscience research Issue: Volume 92:Issue 11(2014:Nov.) Page Start: 1591 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Ex Vivo Gene Therapy: Graft-versus-host Disease (GVHD) in NSG™ (NOD.Cg-Prkdcscid Il2rgtm1Wjl/SzJ) Mice Transplanted with CD34+ Human Hematopoietic Stem Cells. (July 2019) Authors: Chandra, Sundeep; Cristofori, Patrizia; Fonck, Carlos; O'Neill, Charles A. Journal: Toxicologic pathology Issue: Volume 47:Number 5(2019) Page Start: 656 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. Genetic variation in aryl N‐acetyltransferase results in significant differences in the pharmacokinetic and safety profiles of amifampridine (3, 4‐diaminopyridine) phosphate. Issue 1 (9th December 2014) Authors: Haroldsen, Peter E.; Garovoy, Marvin R.; Musson, Donald G.; Zhou, Huiyu; Tsuruda, Laurie; Hanson, Boyd; O'Neill, Charles A. Journal: Pharmacology research & perspectives Issue: Volume 3:Issue 1(2015:Feb.) Page Start: n/a Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. Intravitreal enzyme replacement inhibits progression of retinal degeneration in canine CLN2 neuronal ceroid lipofuscinosis. (September 2020) Authors: Whiting, Rebecca E.H.; Robinson Kick, Grace; Ota-Kuroki, Juri; Lim, Stefanie; Castaner, Leilani J.; Jensen, Cheryl A.; Kowal, Joseph; Nguyen, Annalisa; Corado, Carley; O'Neill, Charles A.; Katz, Martin L. Journal: Experimental eye research Issue: Volume 198(2020) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
6. Intravitreal enzyme replacement preserves retinal structure and function in canine CLN2 neuronal ceroid lipofuscinosis. (August 2020) Authors: Whiting, Rebecca E.H.; Pearce, Jacqueline W.; Vansteenkiste, Daniella P.; Bibi, Katherine; Lim, Stefanie; Robinson Kick, Grace; Castaner, Leilani J.; Sinclair, John; Chandra, Sundeep; Nguyen, Annalisa; O'Neill, Charles A.; Katz, Martin L. Journal: Experimental eye research Issue: Volume 197(2020) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
7. Workshop Proceedings: Streamlined Development of Safety Assessment Programs Supporting Orphan/Rare Diseases—Are We There Yet?. (July 2016) Authors: Allamneni, Krishna P.; Parker, Suezanne; O'Neill, Charles A.; Wright, Teresa L.; King, Sruthi; Andrews, Laura Journal: International journal of toxicology Issue: Volume 35:Number 4(2016) Page Start: 393 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗