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dc.contributor.authorGiugliani, Robertopt_BR
dc.contributor.authorMartins, Ana Maria (Medicina)pt_BR
dc.contributor.authorOkuyama, Torayukipt_BR
dc.contributor.authorEto, Yoshikatsupt_BR
dc.contributor.authorSakai, Noriopt_BR
dc.contributor.authorNakamura, Kimitoshipt_BR
dc.contributor.authorMorimoto, Hidetopt_BR
dc.contributor.authorMinami, Kohtatopt_BR
dc.contributor.authorYamamoto, Tatsuyoshipt_BR
dc.contributor.authorYamaoka, Marikopt_BR
dc.contributor.authorIkeda, Toshiakipt_BR
dc.contributor.authorSo, Saireipt_BR
dc.contributor.authorTanizawa, Kazunoript_BR
dc.contributor.authorSonoda, Hiroyukipt_BR
dc.contributor.authorSchmidt, Mathiaspt_BR
dc.contributor.authorSato, Yujipt_BR
dc.date.accessioned2022-01-27T04:31:56Zpt_BR
dc.date.issued2021pt_BR
dc.identifier.issn1422-0067pt_BR
dc.identifier.urihttp://hdl.handle.net/10183/234494pt_BR
dc.description.abstractEnzyme replacement therapy (ERT) improves somatic manifestations in mucopolysaccharidoses (MPS). However, because intravenously administered enzymes cannot cross the blood–brain barrier (BBB), ERT is ineffective against the progressive neurodegeneration and resultant severe central nervous system (CNS) symptoms observed in patients with neuronopathic MPS. Attempts to surmount this problem have been made with intrathecal and intracerebroventricular ERT in order to achieve CNS effects, but the burdens on patients are inimical to long-term administrations. However, since pabinafusp alfa, a human iduronate-2-sulfatase fused with a BBB-crossing anti-transferrin receptor antibody, showed both central and peripheral efficacy in a mouse model, subsequent clinical trials in a total of 62 patients with MPS-II (Hunter syndrome) in Japan and Brazil substantiated this dual efficacy and provided an acceptable safety profile. To date, pabinafusp alfa is the only approved intravenous ERT that is effective against both the somatic and CNS symptoms of patients with MPS-II. This article summarizes the previously obtained preclinical and clinical evidence related to the use of this drug, presents latest data, and discusses the preclinical, translational, and clinical challenges of evaluating, ameliorating, and preventing neurodegeneration in patients with MPS-II.en
dc.format.mimetypeapplication/pdfpt_BR
dc.language.isoengpt_BR
dc.relation.ispartofInternational journal of molecular sciences. Basel. Vol. 22 (2021), 10938, 15 p.pt_BR
dc.rightsOpen Accessen
dc.subjectNeuronopathic mucopolysaccharidosisen
dc.subjectMucopolissacaridose IIpt_BR
dc.subjectTerapia de reposição de enzimaspt_BR
dc.subjectHunter syndromeen
dc.subjectMucopolysaccharidosis IIen
dc.subjectIduronato sulfatasept_BR
dc.subjectIduronate-2-sulfataseen
dc.subjectDisfunção cognitivapt_BR
dc.subjectEnzyme replacement therapyen
dc.subjectNeurodegenerationen
dc.subjectNeurocognitive impairmenten
dc.subjectPabinafusp alfaen
dc.subjectBlood–brain barrieren
dc.titleEnzyme replacement therapy with pabinafusp alfa for neuronopathic mucopolysaccharidosis II : an integrated analysis of preclinical and clinical datapt_BR
dc.typeArtigo de periódicopt_BR
dc.identifier.nrb001136217pt_BR
dc.type.originEstrangeiropt_BR


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