NOVEL SMALL MOLECULE THERAPY FOR MUCOPOLYSACCHARIDOSIS TYPE I

被引：0
|
作者：
Bodamer, Olaf [1 ,2 ]
Brothers, Shaun [3 ,4 ]
Volmar, Claude-Henry [3 ,4 ]
Wahlestedt, Claes [3 ,4 ]
机构：
[1] Boston Childrens Hosp, BoLD Boston Childrens Lysosomal Storage Dis Progr, Boston, MA USA
[2] Boston Childrens Hosp, Div Genet & Genom, Boston, MA USA
[3] Univ Miami, Ctr Therapeut Innovat, Miami, FL USA
[4] Univ Miami, Dept Psychiat & Behav Sci, Miami, FL USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2016年 / 117卷 / 03期
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：
21
引用
收藏
页码：247 / 247
页数：1
相关论文
共 50 条
  • [21] Mucopolysaccharidosis Type I in a Dog
    Amaral, Andreza Da Silva
    Agassi De Sales, Nathali Adrielli
    Rosado, Isabel Rodrigues
    Giugliani, Roberto
    Burin, Maira Graeff
    Baldo, Guilherme
    Martin, Ian
    Leonel Alves, Endrigo Gabellini
    ACTA SCIENTIAE VETERINARIAE, 2021, 49
  • [22] Mucopolysaccharidosis type I in Taiwan
    Lin, SP
    Lee-Chen, GJ
    Chuang, CK
    Hwu, WL
    Kuo, MT
    Yang, C
    EUROPEAN JOURNAL OF HUMAN GENETICS, 2002, 10 : 209 - 209
  • [23] Enzyme replacement therapy in mucopolysaccharidosis type I: Progress and emerging difficulties
    Wraith, JE
    JOURNAL OF INHERITED METABOLIC DISEASE, 2001, 24 (02) : 245 - 250
  • [24] Neonatal gene therapy in a Mucopolysaccharidosis type I (MPSI) mouse model
    De Ponti, G.
    Santi, L.
    Dina, G.
    Pievani, A.
    Donsante, S.
    Riminucci, M.
    Corsi, A.
    Sawamoto, K.
    Passerini, L.
    Annoni, A.
    Gregori, S.
    Biondi, A.
    Quattrini, A.
    Tomatsu, S.
    Aiuti, A.
    Bernardo, M. E.
    Serafini, M.
    HUMAN GENE THERAPY, 2022, 33 (23-24) : A171 - A172
  • [25] Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I
    Jameson, Elisabeth
    Jones, Simon
    Wraith, James E.
    COCHRANE DATABASE OF SYSTEMATIC REVIEWS, 2013, (11):
  • [26] Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I
    Jameson, Elisabeth
    Jones, Simon
    Wraith, James E.
    COCHRANE DATABASE OF SYSTEMATIC REVIEWS, 2013, (09):
  • [27] Cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I
    Braunlin, Elizabeth A.
    Berry, James M.
    Whitley, Chester B.
    AMERICAN JOURNAL OF CARDIOLOGY, 2006, 98 (03): : 416 - 418
  • [28] Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I
    Jameson, E.
    Jones, S.
    Remmington, T.
    COCHRANE DATABASE OF SYSTEMATIC REVIEWS, 2019, (06):
  • [29] Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I
    Jameson, Elisabeth
    Jones, Simon
    Remmington, Tracey
    COCHRANE DATABASE OF SYSTEMATIC REVIEWS, 2016, (04):
  • [30] Induction of immune tolerance to enzyme replacement therapy in mucopolysaccharidosis type I
    Liao, Ai Yin
    Ghosh, Arunabha
    O'Leary, Claire
    Mercer, Jean
    Church, Heather J.
    Tylee, Karen L.
    Goenka, Anu
    Holley, Rebecca
    Wynn, Robert F.
    Jones, Simon A.
    Bigger, Brian W.
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : S86 - S86
12345