Navegação Ciências Biológicas por Assunto "iduronate-2-sulfatase"

Resultados 1-3 de 3

    • Enzyme replacement therapy with pabinafusp alfa for neuronopathic mucopolysaccharidosis II : an integrated analysis of preclinical and clinical data 

      Giugliani, Roberto; Martins, Ana Maria (Medicina); Okuyama, Torayuki; Eto, Yoshikatsu; Sakai, Norio; Nakamura, Kimitoshi; Morimoto, Hideto; Minami, Kohtato; Yamamoto, Tatsuyoshi; Yamaoka, Mariko; Ikeda, Toshiaki; So, Sairei; Tanizawa, Kazunori; Sonoda, Hiroyuki; Schmidt, Mathias; Sato, Yuji (2021) [Artigo de periódico]
      Enzyme 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 ...

    • Guidelines for diagnosis and treatment of Hunter Syndrome for clinicians in Latin America 

      Giugliani, Roberto; Solano Villareal, Martha Luz; Arellano Valdez, Carmen Araceli; Mahfoud Hawilou, Antonieta; Guelbert, Norberto; Correa Garzón, Luz Norela; Martins, Ana Maria (Medicina); Acosta, Angelina Xavier; Cabello, Juan Francisco; Lemes, Aída; Santos, Mara Lúcia Schmitz Ferreira; Amartino, Hernan (2014) [Artigo de periódico]
      This review aims to provide clinicians in Latin America with the most current information on the clinical aspects, diagnosis, and management of Hunter syndrome, a serious and progressive disease for which specific treatment ...

    • Iduronate-2-sulfatase fused with anti-hTfR antibody, pabinafusp alfa, for MPS-II : a phase 2 trial in Brazil 

      Giugliani, Roberto; Martins, Ana Maria (Medicina); So, Sairei; Yamamoto, Tatsuyoshi; Yamaoka, Mariko; Ikeda, Toshiaki; Tanizawa, Kazunori; Sonoda, Hiroyuki; Schmidt, Mathias; Sato, Yuji (2021) [Artigo de periódico]
      In Hunter syndrome (mucopolysaccharidosis II [MPS-II]),systemic accumulation of glycosaminoglycans (GAGs) dueto a deficiency of iduronate-2-sulfatase (IDS), caused by mu-tations in theIDSgene, leads to multiple somatic ...