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Mucopolysaccharidosis VII in Brazil: natural history and clinical findings.
Giugliani R, Barth AL, Dumas MRC, da Silva Franco JF, de Rosso Giuliani L, Grangeiro CHP, Horovitz DDG, Kim CA, de Araújo Leão EKE, de Medeiros PFV, Miguel DSCG, Moreira MESA, Dos Santos HMGP, da Silva LCS, da Silva LR, de Souza IN, Nalin T, Garcia D. Giugliani R, et al. Among authors: horovitz ddg. Orphanet J Rare Dis. 2021 May 22;16(1):238. doi: 10.1186/s13023-021-01870-w. Orphanet J Rare Dis. 2021. PMID: 34022924 Free PMC article. Review.
Enzyme replacement therapy for Mucopolysaccharidosis Type I among patients followed within the MPS Brazil Network.
Dornelles AD, de Camargo Pinto LL, de Paula AC, Steiner CE, Lourenço CM, Kim CA, Horovitz DD, Ribeiro EM, Valadares ER, Goulart I, Neves de Souza IC, da Costa Neri JI, Santana-da-Silva LC, Silva LR, Ribeiro M, de Oliveira Sobrinho RP, Giugliani R, Schwartz IV. Dornelles AD, et al. Genet Mol Biol. 2014 Mar;37(1):23-9. doi: 10.1590/s1415-47572014000100006. Epub 2013 Feb 28. Genet Mol Biol. 2014. PMID: 24688287 Free PMC article.
Clinical Characterization of Mucolipidoses II and III: A Multicenter Study.
Alegra T, Sperb-Ludwig F, Guarany NR, Ribeiro EM, Lourenço CM, Kim CA, Valadares ER, Galera MF, Acosta AX, Horovitz DDG, Schwartz IVD. Alegra T, et al. Among authors: horovitz ddg. J Pediatr Genet. 2019 Dec;8(4):198-204. doi: 10.1055/s-0039-1697605. Epub 2019 Sep 24. J Pediatr Genet. 2019. PMID: 31687257 Free PMC article.
Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series.
Horovitz DD, Acosta AX, Giugliani R, Hlavatá A, Hlavatá K, Tchan MC, Lopes Barth A, Cardoso L Jr, Embiruçu de Araújo Leão EK, Esposito AC, Kyosen SO, De Souza CF, Martins AM. Horovitz DD, et al. Orphanet J Rare Dis. 2016 Apr 29;11(1):51. doi: 10.1186/s13023-016-0437-8. Orphanet J Rare Dis. 2016. PMID: 27129473 Free PMC article.
56 results