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Journal of Huntington'S Disease|October 3, 2022
Brain Alterations in Aged OVT73 Sheep Model of Huntington's Disease: An MRI Based ApproachToloo Taghian, Jillian Gallagher, Erin Batcho, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 3, 2018
Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic MiceStuart Bunting, Lening Zhang, Lin Xie, et al.
Investigative Ophthalmology & Visual Science|February 19, 2011
The pharmacologic assessment of a novel lymphocyte function-associated antigen-1 antagonist (SAR 1118) for the treatment of keratoconjunctivitis sicca in dogsChristopher J Murphy, Ellison Bentley, Paul E Miller, et al.
Toxicology Reports|March 16, 2023
Safety, pharmacokinetics and CNS distribution of tralesinidase alfa administered via intracerebroventricular infusion to juvenile cynomolgus monkeysJason Pinkstaff, Emma McCullagh, Anita Grover, et al.
Journal of Ocular Pharmacology and Therapeutics : the Official Journal of the Association for Ocular Pharmacology and Therapeutics|July 14, 2026
Preexisting Fundus Findings in a Large Cohort of Young Cynomolgus Macaques (Macaca fascicularis) Bred for Ocular Research: Comparison to HumansT Michael Nork, Peter J Sonnentag, Paul E Miller, et al.
Molecular Genetics and Metabolism|September 27, 2014
Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosisBrian R Vuillemenot, Derek Kennedy, Jonathan D Cooper, et al.
Drug Delivery and Translational Research|January 17, 2020
Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type BAnita Grover, Danielle Crippen-Harmon, Lacey Nave, et al.
The Journal of Pharmacology and Experimental Therapeutics|June 18, 2022
Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIBN Matthew Ellinwood, Bethann N Valentine, Andrew S Hess, et al.
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