MUCOPOLYSACCHARIDOSIS TYPE I REPORT OF A SUCCESSFUL HEMATOPOIETIC STEM CELL TRANSPLANTATION AFTER 11 MONTHS OF ENZYME REPLACEMENT THERAPY

被引:0
|
作者
Bittar, C. M. [1 ]
Vairo, F. [1 ]
Souza, C. F. M. [1 ]
Netto, C. B. [1 ]
Gregianin, L. [2 ]
Galvao, C. [2 ]
Fagondes, S. [3 ]
Giugliani, R. [1 ]
机构
[1] HCPA, Med Genet Serv, Porto Alegre, RS, Brazil
[2] HCPA, Oncol Serv, Porto Alegre, RS, Brazil
[3] HCPA, Serv Pneumol, Porto Alegre, RS, Brazil
关键词
D O I
暂无
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
引用
收藏
页码:S220 / S220
页数:1
相关论文
共 50 条
  • [31] Hematopoietic stem cell transplantation for patients with mucopolysaccharidosis type II
    Kubaski, Francyne
    Suzuki, Yasuyuki
    Yabe, Hiromasa
    Mason, Robert W.
    Xie, Li
    Onsten, Tor G. H.
    Leistner-Segal, Sandra
    Giugliani, Roberto
    Dung, Vu C.
    Ngoc, Can T. B.
    Ngoc, Can T. B.
    Yamaguchi, Seiji
    Montano, Adriana M.
    Orii, Kenji E.
    Fukao, Toshiyuki
    Shinataku, Haruo
    Orii, Tadao
    Tomatsu, Shunji
    MOLECULAR GENETICS AND METABOLISM, 2017, 120 (1-2) : S77 - S77
  • [32] Laronidase (Aldurazyme®):: enzyme replacement therapy for mucopolysaccharidosis type I
    Pastores, Gregory M.
    EXPERT OPINION ON BIOLOGICAL THERAPY, 2008, 8 (07) : 1003 - 1009
  • [33] Enzyme replacement therapy in mucopolysaccharidosis type I: The sooner the better
    Coppa, Giovanni V.
    Baldoni, Irene
    Bruni, Stefano
    Gabrielli, Orazio
    CLINICAL THERAPEUTICS, 2007, 29 : S83 - S83
  • [34] A Case of a Bilateral Cicatricial Upper Eyelid Entropion After Hematopoietic Stem Cell Transplantation in Mucopolysaccharidosis Type I
    Dulz, Simon
    Wagenfeld, Lars
    Richard, Gisbert
    Schrum, Johanna
    Muschol, Nicole
    Keserue, Matthias
    OPHTHALMIC PLASTIC AND RECONSTRUCTIVE SURGERY, 2017, 33 (03): : S75 - S77
  • [35] Successful pregnancy and breastfeeding in a woman with mucopolysaccharidosis type I while receiving laronidase enzyme replacement therapy
    Castorina, M.
    Antuzzi, D.
    Richards, S. M.
    Cox, G. F.
    Xue, Y.
    CLINICAL AND EXPERIMENTAL OBSTETRICS & GYNECOLOGY, 2015, 42 (01): : 108 - 113
  • [36] Successful induction of immune tolerance to enzyme replacement therapy in canine mucopolysaccharidosis I
    Kakkis, E
    Lester, T
    Yang, R
    Tanaka, C
    Anand, V
    Lemontt, J
    Peinovich, M
    Passage, M
    PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2004, 101 (03) : 829 - 834
  • [37] Hematopoietic stem cell gene therapy for mucopolysaccharidosis type IIIC
    Badell-Grau, Rafael
    Pakravesh, Kasra
    Sivakumar, Anusha
    Losay, Pauline
    Duong, Kalvin
    Tran, Christine
    Pithia, Rushil
    Khare, Veenita
    Thai, Kevin
    Cherqui, Stephanie
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 13 - 13
  • [38] Hematopoietic Stem Cell Gene Therapy For Mucopolysaccharidosis Type IIIC
    Badell-Grau, Rafael A.
    Rainaldi, Joseph
    Losay, Pauline
    Doung, Kalvin
    Pithia, Rushil
    Pakravesh, Kasra
    Silva, Alexander
    Thai, Eric
    Sharma, Jay
    Paulchakrabarti, Mousumi
    Khare, Veenita
    Cherqui, Stephanie
    MOLECULAR THERAPY, 2022, 30 (04) : 300 - 301
  • [39] Musculoskeletal manifestations in mucopolysaccharidosis type I (Hurler syndrome) following hematopoietic stem cell transplantation
    Mona Schmidt
    Sandra Breyer
    Ulrike Löbel
    Sinef Yarar
    Ralf Stücker
    Kurt Ullrich
    Ingo Müller
    Nicole Muschol
    Orphanet Journal of Rare Diseases, 11
  • [40] Musculoskeletal manifestations in mucopolysaccharidosis type I (Hurler syndrome) following hematopoietic stem cell transplantation
    Schmidt, Mona
    Breyer, Sandra
    Loebel, Ulrike
    Yarar, Sinef
    Stuecker, Ralf
    Ullrich, Kurt
    Mueller, Ingo
    Muschol, Nicole
    ORPHANET JOURNAL OF RARE DISEASES, 2016, 11