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
  • [1] Mucopolysaccharidosis type I therapy
    Murillo-Lopez, FH
    ANNALS OF OPHTHALMOLOGY, 2005, 37 (04) : 235 - 236
  • [2] Novel small molecule therapy development for MPS I
    Brothers, Shaun
    Volmar, Claude-Henry
    Bodamer, Olaf
    Hayward, Marshall
    Wahlestedt, Claes
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : S26 - S26
  • [3] The potential of gene therapy for mucopolysaccharidosis type I
    Pimentel Vera, Luisa Natalia
    Baldo, Guilherme
    EXPERT OPINION ON ORPHAN DRUGS, 2020, 8 (01): : 33 - 41
  • [4] Enzyme replacement therapy in mucopolysaccharidosis type I
    Miebach, E
    ACTA PAEDIATRICA, 2005, 94 : 58 - 60
  • [5] SMALL MOLECULE INHIBITORS OF GLYCOSAMINOGLYCAN BIOSYNTHESIS AS A SUBSTRATE REDUCTION THERAPY FOR MUCOPOLYSACCHARIDOSIS
    Brown, J. R.
    Witt, R.
    Carroll, R.
    Glass, C.
    Crawford, B.
    MOLECULAR GENETICS AND METABOLISM, 2009, 98 (1-2) : 64 - 64
  • [6] Small Molecule Inhibitors of Glycosaminoglycan Biosynthesis as a Substrate Reduction Therapy for Mucopolysaccharidosis
    Brown, Jillian
    Witt, Robert
    Carroll, Raleina
    Glass, Charles
    Crawford, Brett
    GLYCOBIOLOGY, 2009, 19 (11) : 1322 - 1322
  • [7] Enzyme replacement therapy for mucopolysaccharidosis type I in Japan
    Okuyama, T.
    Tanaka, T.
    JOURNAL OF INHERITED METABOLIC DISEASE, 2008, 31 : 119 - 119
  • [8] An adenoviral mediated gene therapy for mucopolysaccharidosis type I
    Hurt, Sarah
    Le, Steven Q.
    Mendonca, Samir
    Dickson, Patricia
    Curiel, David T.
    MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S60 - S60
  • [9] Mucopolysaccharidosis Type I
    Wraith, J. E.
    Jones, Simon
    PEDIATRIC ENDOCRINOLOGY REVIEWS PER, 2014, 12 : 102 - 106
  • [10] Mucopolysaccharidosis Type I
    Kubaski, Francyne
    Poswar, Fabiano de Oliveira
    Michelin-Tirelli, Kristiane
    Matte, Ursula da Silveira
    Horovitz, Dafne D.
    Barth, Anneliese Lopes
    Baldo, Guilherme
    Vairo, Filippo
    Giugliani, Roberto
    DIAGNOSTICS, 2020, 10 (03)
12345