Navegação por Assunto "Alpha-L-iduronidase"

Resultados 1-2 de 2

    • Enzyme replacement therapy for mucopolysaccharidosis type I among patients followed within the MPS Brazil network 

      Dornelles, Alícia Dorneles; Pinto, Louise Lapagesse de Camargo; Paula, Ana Carolina de; Steiner, Carlos Eduardo; Lourenço, Charles Marques; Kim, Chong Ae; Horowitz, Dafne Dain Gandelman; Ribeiro, Erlane Marques; Valadares, Eugênia Ribeiro; Goulart, Isabela; Souza, Isabel Cristina Neves de; Neri, João Ivanildo da Costa; Silva, Luiz Carlos Santana da; Silva, Luiz Roberto da; Ribeiro, Márcia Gonçalves; Oliveira Sobrinho, Ruy Pires de; Giugliani, Roberto; Schwartz, Ida Vanessa Doederlein (2014) [Artigo de periódico]
      Mucopolysaccharidosis type I (MPS I) is a rare lysosomal disorder caused by deficiency of alpha-L-iduronidase. Few clinical trials have assessed the effect of enzyme replacement therapy (ERT) for this condition. We conducted ...

    • Mucopolissacaridose tipo I em cão 

      Amaral, Andreza da Silva; Sales, Nathali Adrielli Agassi de; Rosado, Isabel Rodrigues; Giugliani, Roberto; Burin, Maira Graeff; Baldo, Guilherme; Martin, Ian; Alves, Endrigo Gabellini Leonel (2021) [Artigo de periódico]
      Background: Mucopolysaccharidoses (MPS) are a group of rare illnesses caused by deficient activity of enzymes required for degradation of glycosaminoglycans (GAGs). Each type of MPS is caused by mutations in one of the ...