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Mucopolysaccharidosis VII in Brazil: natural history and clinical findings.
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.
The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII.
Wang RY, da Silva Franco JF, López-Valdez J, Martins E, Sutton VR, Whitley CB, Zhang L, Cimms T, Marsden D, Jurecka A, Harmatz P.
Wang RY, et al. Among authors: da silva franco jf.
Mol Genet Metab. 2020 Mar;129(3):219-227. doi: 10.1016/j.ymgme.2020.01.003. Epub 2020 Jan 11.
Mol Genet Metab. 2020.
PMID: 32063397
Free article.
Clinical Trial.
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Corrigendum to "The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII" [Mol Genet Metab 2020 Mar;129(3):219-227].
Wang RY, da Silva Franco JF, López-Valdez J, Martins E, Sutton VR, Whitley CB, Zhang L, Cimms T, Marsden D, Jurecka A, Harmatz P.
Wang RY, et al. Among authors: da silva franco jf.
Mol Genet Metab. 2020 Sep-Oct;131(1-2):285. doi: 10.1016/j.ymgme.2020.08.001. Epub 2020 Aug 23.
Mol Genet Metab. 2020.
PMID: 32843286
No abstract available.
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Thrombomodulin gene mutation and associated predisposing factors in familial collapsing glomerulopathy.
Riguetti MTP, Varela-Calais P, Fernandes DE, da Silva Franco JF, Ribeiro Nogueira B, Pesquero JB, Mastroianni-Kirsztajn G.
Riguetti MTP, et al. Among authors: da silva franco jf.
Nephron. 2024 Feb 19. doi: 10.1159/000536244. Online ahead of print.
Nephron. 2024.
PMID: 38373411
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