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Final results of the phase 1/2, open-label clinical study of intravenous recombinant human N-acetyl-α-d-glucosaminidase (SBC-103) in children with mucopolysaccharidosis IIIB.
Whitley CB, Vijay S, Yao B, Pineda M, Parker GJM, Rojas-Caro S, Zhang X, Dai Y, Cinar A, Bubb G, Patki KC, Escolar ML. Whitley CB, et al. Among authors: rojas caro s. Mol Genet Metab. 2019 Feb;126(2):131-138. doi: 10.1016/j.ymgme.2018.12.003. Epub 2018 Dec 6. Mol Genet Metab. 2019. PMID: 30635159 Clinical Trial.
Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study.
Jones SA, Rojas-Caro S, Quinn AG, Friedman M, Marulkar S, Ezgu F, Zaki O, Gargus JJ, Hughes J, Plantaz D, Vara R, Eckert S, Arnoux JB, Brassier A, Le Quan Sang KH, Valayannopoulos V. Jones SA, et al. Among authors: rojas caro s. Orphanet J Rare Dis. 2017 Feb 8;12(1):25. doi: 10.1186/s13023-017-0587-3. Orphanet J Rare Dis. 2017. PMID: 28179030 Free PMC article. Clinical Trial.
A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency.
Burton BK, Balwani M, Feillet F, Barić I, Burrow TA, Camarena Grande C, Coker M, Consuelo-Sánchez A, Deegan P, Di Rocco M, Enns GM, Erbe R, Ezgu F, Ficicioglu C, Furuya KN, Kane J, Laukaitis C, Mengel E, Neilan EG, Nightingale S, Peters H, Scarpa M, Schwab KO, Smolka V, Valayannopoulos V, Wood M, Goodman Z, Yang Y, Eckert S, Rojas-Caro S, Quinn AG. Burton BK, et al. Among authors: rojas caro s. N Engl J Med. 2015 Sep 10;373(11):1010-20. doi: 10.1056/NEJMoa1501365. N Engl J Med. 2015. PMID: 26352813 Free article. Clinical Trial.
Quality-controlled measurement methods for quantification of variations in transcript abundance in whole blood samples from healthy volunteers.
Peters EH, Rojas-Caro S, Brigell MG, Zahorchak RJ, des Etages SA, Ruppel PL, Knight CR, Austermiller B, Graham MC, Wowk S, Banks S, Madabusi LV, Turk P, Wilder D, Kempfer C, Osborn TW, Willey JC. Peters EH, et al. Among authors: rojas caro s. Clin Chem. 2007 Jun;53(6):1030-7. doi: 10.1373/clinchem.2006.078154. Epub 2007 Apr 13. Clin Chem. 2007. PMID: 17434908
The G0/G1 switch gene 2 is a novel PPAR target gene.
Zandbergen F, Mandard S, Escher P, Tan NS, Patsouris D, Jatkoe T, Rojas-Caro S, Madore S, Wahli W, Tafuri S, Müller M, Kersten S. Zandbergen F, et al. Among authors: rojas caro s. Biochem J. 2005 Dec 1;392(Pt 2):313-24. doi: 10.1042/BJ20050636. Biochem J. 2005. PMID: 16086669 Free PMC article.