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Page 1
Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB.
Okur I, Ezgu F, Giugliani R, Muschol N, Koehn A, Amartino H, Harmatz P, de Castro Lopez MJ, Couce ML, Lin SP, Batzios S, Cleary M, Solano M, Peters H, Lee J, Nestrasil I, Shaywitz AJ, Maricich SM, Kuca B, Kovalchin J, Zanelli E. Okur I, et al. Among authors: nestrasil i. J Pediatr. 2022 Oct;249:50-58.e2. doi: 10.1016/j.jpeds.2022.06.005. Epub 2022 Jun 13. J Pediatr. 2022. PMID: 35709957 Free article.
Cognitive, medical, and neuroimaging characteristics of attenuated mucopolysaccharidosis type II.
Yund B, Rudser K, Ahmed A, Kovac V, Nestrasil I, Raiman J, Mamak E, Harmatz P, Steiner R, Lau H, Vekaria P, Wozniak JR, Lim KO, Delaney K, Whitley C, Shapiro EG. Yund B, et al. Among authors: nestrasil i. Mol Genet Metab. 2015 Feb;114(2):170-7. doi: 10.1016/j.ymgme.2014.12.299. Epub 2014 Dec 9. Mol Genet Metab. 2015. PMID: 25541100 Free PMC article. Clinical Trial.
Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment.
Shapiro EG, Nestrasil I, Rudser K, Delaney K, Kovac V, Ahmed A, Yund B, Orchard PJ, Eisengart J, Niklason GR, Raiman J, Mamak E, Cowan MJ, Bailey-Olson M, Harmatz P, Shankar SP, Cagle S, Ali N, Steiner RD, Wozniak J, Lim KO, Whitley CB. Shapiro EG, et al. Among authors: nestrasil i. Mol Genet Metab. 2015 Sep-Oct;116(1-2):61-8. doi: 10.1016/j.ymgme.2015.06.002. Epub 2015 Jun 17. Mol Genet Metab. 2015. PMID: 26095521 Free PMC article. Clinical Trial.
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIA.
Shapiro EG, Nestrasil I, Delaney KA, Rudser K, Kovac V, Nair N, Richard CW 3rd, Haslett P, Whitley CB. Shapiro EG, et al. Among authors: nestrasil i. J Pediatr. 2016 Mar;170:278-87.e1-4. doi: 10.1016/j.jpeds.2015.11.079. Epub 2016 Jan 16. J Pediatr. 2016. PMID: 26787381 Free PMC article.
Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial.
Giugliani R, Giugliani L, de Oliveira Poswar F, Donis KC, Corte AD, Schmidt M, Boado RJ, Nestrasil I, Nguyen C, Chen S, Pardridge WM. Giugliani R, et al. Among authors: nestrasil i. Orphanet J Rare Dis. 2018 Jul 5;13(1):110. doi: 10.1186/s13023-018-0849-8. Orphanet J Rare Dis. 2018. PMID: 29976218 Free PMC article. Clinical Trial.
Attention and corpus callosum volumes in individuals with mucopolysaccharidosis type I.
King KE, Rudser KD, Nestrasil I, Kovac V, Delaney KA, Wozniak JR, Mueller BA, Lim KO, Eisengart JB, Mamak EG, Raiman J, Ali N, Cagle S, Harmatz P, Whitley CB, Shapiro EG. King KE, et al. Among authors: nestrasil i. Neurology. 2019 May 14;92(20):e2321-e2328. doi: 10.1212/WNL.0000000000007496. Epub 2019 Apr 12. Neurology. 2019. PMID: 30979856 Free PMC article.
Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study.
Chen AH, Harmatz P, Nestrasil I, Eisengart JB, King KE, Rudser K, Kaizer AM, Svatkova A, Wakumoto A, Le SQ, Madden J, Young S, Zhang H, Polgreen LE, Dickson PI. Chen AH, et al. Among authors: nestrasil i. Mol Genet Metab. 2020 Feb;129(2):80-90. doi: 10.1016/j.ymgme.2019.11.007. Epub 2019 Nov 30. Mol Genet Metab. 2020. PMID: 31839529 Free PMC article. Clinical Trial.
Quantitative brain MRI morphology in severe and attenuated forms of mucopolysaccharidosis type I.
Kovac V, Shapiro EG, Rudser KD, Mueller BA, Eisengart JB, Delaney KA, Ahmed A, King KE, Yund BD, Cowan MJ, Raiman J, Mamak EG, Harmatz PR, Shankar SP, Ali N, Cagle SR, Wozniak JR, Lim KO, Orchard PJ, Whitley CB, Nestrasil I. Kovac V, et al. Among authors: nestrasil i. Mol Genet Metab. 2022 Feb;135(2):122-132. doi: 10.1016/j.ymgme.2022.01.001. Epub 2022 Jan 7. Mol Genet Metab. 2022. PMID: 35012890 Free PMC article.
Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB.
Ellinwood NM, Valentine BN, Hess AS, Jens JK, Snella EM, Jamil M, Hostetter SJ, Jeffery ND, Smith JD, Millman ST, Parsons RL, Butt MT, Chandra S, Egeland MT, Assis AB, Nelvagal HR, Cooper JD, Nestrasil I, Mueller BA, Labounek R, Paulson A, Prill H, Liu XY, Zhou H, Lawrence R, Crawford BE, Grover A, Cherala G, Melton AC, Cherukuri A, Vuillemenot BR, Wait JCM, O'Neill CA, Pinkstaff J, Kovalchin J, Zanelli E, McCullagh E. Ellinwood NM, et al. Among authors: nestrasil i. J Pharmacol Exp Ther. 2022 Sep;382(3):277-286. doi: 10.1124/jpet.122.001119. Epub 2022 Jun 18. J Pharmacol Exp Ther. 2022. PMID: 35717448 Free PMC article.
61 results