O'Neill CA - Search Results

1

Enzyme replacement therapy delays pupillary light reflex deficits in a canine model of late infantile neuronal ceroid lipofuscinosis.

Whiting RE, Narfström K, Yao G, Pearce JW, Coates JR, Castaner LJ, Jensen CA, Dougherty BN, Vuillemenot BR, Kennedy D, O'Neill CA, Katz ML. Whiting RE, et al. Exp Eye Res. 2014 Aug;125:164-72. doi: 10.1016/j.exer.2014.06.008. Epub 2014 Jun 19. Exp Eye Res. 2014. PMID: 24954537 Free article.

2

Intrathecal tripeptidyl-peptidase 1 reduces lysosomal storage in a canine model of late infantile neuronal ceroid lipofuscinosis.

Vuillemenot BR, Katz ML, Coates JR, Kennedy D, Tiger P, Kanazono S, Lobel P, Sohar I, Xu S, Cahayag R, Keve S, Koren E, Bunting S, Tsuruda LS, O'Neill CA. Vuillemenot BR, et al. Mol Genet Metab. 2011 Nov;104(3):325-37. doi: 10.1016/j.ymgme.2011.06.018. Epub 2011 Jun 30. Mol Genet Metab. 2011. PMID: 21784683

3

Recombinant human tripeptidyl peptidase-1 infusion to the monkey CNS: safety, pharmacokinetics, and distribution.

Vuillemenot BR, Kennedy D, Reed RP, Boyd RB, Butt MT, Musson DG, Keve S, Cahayag R, Tsuruda LS, O'Neill CA. Vuillemenot BR, et al. Toxicol Appl Pharmacol. 2014 May 15;277(1):49-57. doi: 10.1016/j.taap.2014.03.005. Epub 2014 Mar 15. Toxicol Appl Pharmacol. 2014. PMID: 24642058

4

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

Katz ML, Coates JR, Sibigtroth CM, Taylor JD, Carpentier M, Young WM, Wininger FA, Kennedy D, Vuillemenot BR, O'Neill CA. Katz ML, et al. J Neurosci Res. 2014 Nov;92(11):1591-8. doi: 10.1002/jnr.23423. Epub 2014 Jun 17. J Neurosci Res. 2014. PMID: 24938720 Free PMC article.

5

Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis.

Vuillemenot BR, Kennedy D, Cooper JD, Wong AM, Sri S, Doeleman T, Katz ML, Coates JR, Johnson GC, Reed RP, Adams EL, Butt MT, Musson DG, Henshaw J, Keve S, Cahayag R, Tsuruda LS, O'Neill CA. Vuillemenot BR, et al. Mol Genet Metab. 2015 Feb;114(2):281-93. doi: 10.1016/j.ymgme.2014.09.004. Epub 2014 Sep 16. Mol Genet Metab. 2015. PMID: 25257657

6

Reveglucosidase alfa (BMN 701), an IGF2-Tagged rhAcid α-Glucosidase, Improves Respiratory Functional Parameters in a Murine Model of Pompe Disease.

Peng J, Dalton J, Butt M, Tracy K, Kennedy D, Haroldsen P, Cahayag R, Zoog S, O'Neill CA, Tsuruda LS. Peng J, et al. J Pharmacol Exp Ther. 2017 Feb;360(2):313-323. doi: 10.1124/jpet.116.235952. Epub 2016 Nov 16. J Pharmacol Exp Ther. 2017. PMID: 27856936

7

Immunogenicity to cerliponase alfa intracerebroventricular enzyme replacement therapy for CLN2 disease: Results from a Phase 1/2 study.

Cherukuri A, Cahan H, de Hart G, Van Tuyl A, Slasor P, Bray L, Henshaw J, Ajayi T, Jacoby D, O'Neill CA, Schweighardt B. Cherukuri A, et al. Clin Immunol. 2018 Dec;197:68-76. doi: 10.1016/j.clim.2018.09.003. Epub 2018 Sep 8. Clin Immunol. 2018. PMID: 30205177 Clinical Trial.

8

Intravitreal enzyme replacement preserves retinal structure and function in canine CLN2 neuronal ceroid lipofuscinosis.

Whiting REH, Pearce JW, Vansteenkiste DP, Bibi K, Lim S, Robinson Kick G, Castaner LJ, Sinclair J, Chandra S, Nguyen A, O'Neill CA, Katz ML. Whiting REH, et al. Exp Eye Res. 2020 Aug;197:108130. doi: 10.1016/j.exer.2020.108130. Epub 2020 Jul 1. Exp Eye Res. 2020. PMID: 32622066 Free PMC article.

9

Intravitreal enzyme replacement inhibits progression of retinal degeneration in canine CLN2 neuronal ceroid lipofuscinosis.

Whiting REH, Robinson Kick G, Ota-Kuroki J, Lim S, Castaner LJ, Jensen CA, Kowal J, Nguyen A, Corado C, O'Neill CA, Katz ML. Whiting REH, et al. Exp Eye Res. 2020 Sep;198:108135. doi: 10.1016/j.exer.2020.108135. Epub 2020 Jul 4. Exp Eye Res. 2020. PMID: 32634395 Free PMC article.

10

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

Hammon K, de Hart G, Vuillemenot BR, Kennedy D, Musson D, O'Neill CA, Katz ML, Henshaw JW. Hammon K, et al. Clin Transl Sci. 2021 Sep;14(5):1810-1821. doi: 10.1111/cts.13028. Epub 2021 Jun 2. Clin Transl Sci. 2021. PMID: 34076336 Free PMC article. Clinical Trial.

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