Impact of enzyme replacement therapy and hematopoietic stem cell therapy on growth in patients with Hunter syndrome

被引:0
作者
Tomatsu, Shunji [1 ]
Patel, Pravin [1 ]
Suzuki, Yasuyuki [2 ]
Yabe, Hiromasa [3 ]
Shimada, Tsutomu [1 ]
Mason, Robert W. [1 ]
Orii, Tadao [2 ]
机构
[1] Nemours Alfred I duPont Hosp Children, Wilmington, DE USA
[2] Gifu Univ, Gifu, Japan
[3] Tokai Univ, Sch Med, Isehara, Kanagawa 25911, Japan
关键词
D O I
10.1016/j.ymgme.2014.12.264
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
261
引用
收藏
页码:S115 / S116
页数:2
相关论文
共 50 条
[31]   Ocular Outcomes of Combination Enzyme Replacement Therapy with Hematopoietic Cell Transplantation in Hurler Syndrome [J].
Decanini, Alejandra ;
Tokarev, Julian ;
Eisengart, Julie ;
Shapiro, Elsa ;
Summers, C. Gail ;
Orchard, Paul ;
Tolar, Jakub ;
Bothun, Erick D. .
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2015, 56 (07)
[32]   Hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT) in a patient with mucopolysaccharidosis type I, Hurler syndrome [J].
McNeil, Colleen ;
Casey, Robin ;
Ferreira, Patrick .
MOLECULAR GENETICS AND METABOLISM, 2007, 92 (04) :S30-S30
[33]   Hypervitaminosis A in Pediatric Hematopoietic Stem Cell Patients Requiring Renal Replacement Therapy [J].
Lipkin, Ann Connell ;
Lenssen, Polly .
NUTRITION IN CLINICAL PRACTICE, 2008, 23 (06) :621-629
[34]   Enzyme replacement therapy in mucopolysaccharidosis type II (Hunter syndrome): a preliminary report [J].
Muenzer, J ;
Lamsa, JC ;
Garcia, A ;
Dacosta, J ;
Garcia, J ;
Treco, DA .
ACTA PAEDIATRICA, 2002, 91 :98-99
[35]   ENZYME REPLACEMENT THERAPY IN HUNTER SYNDROME AND THE INCIDENCE AND TIME OF ONSET OF INFUSION REACTIONS [J].
Burton, B. K. ;
Whiteman, D. A. H. .
MOLECULAR GENETICS AND METABOLISM, 2009, 98 (1-2) :83-83
[36]   The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensus [J].
Joseph Muenzer ;
Olaf Bodamer ;
Barbara Burton ;
Lorne Clarke ;
Gudrun Schulze Frenking ;
Roberto Giugliani ;
Simon Jones ;
Maria Verónica Muñoz Rojas ;
Maurizio Scarpa ;
Michael Beck ;
Paul Harmatz .
European Journal of Pediatrics, 2012, 171 :181-188
[37]   The one year experience of enzyme replacement therapy for mucopolysaccharidosis II (Hunter syndrome). [J].
Muenzer, J ;
Calikoglu, M ;
Towle, D ;
McCandless, S ;
Kimura, A .
AMERICAN JOURNAL OF HUMAN GENETICS, 2003, 73 (05) :623-623
[38]   Idursulfase Enzyme Replacement Therapy in Seriously Ill, Japanese Men with Hunter Syndrome [J].
Okuyama, T. ;
Tanaka, A. ;
Tanaka, T. ;
Ida, H. ;
Suzuki, Y. ;
Cox, G. F. ;
Eto, Y. ;
Orii, T. .
MOLECULAR GENETICS AND METABOLISM, 2009, 98 (1-2) :65-65
[39]   COMBINED ENZYME REPLACEMENT THERAPY AND HEMATOPOIETIC STEM CELL TRANSPLANTATION IN MUCOPOLYSACCHARIDOSIS I: THE MANCHESTER EXPERIENCE [J].
Mercer, J. ;
Tylee, K. ;
Page, J. ;
Jones, S. ;
Wraith, E. ;
Wynn, R. .
CLINICAL THERAPEUTICS, 2008, 30 :S123-S123
[40]   Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease [J].
Potter, Jane E. ;
Petts, Gemma ;
Ghosh, Arunabha ;
White, Fiona J. ;
Kinsella, Jane L. ;
Hughes, Stephen ;
Roberts, Jane ;
Hodgkinson, Adam ;
Brammeier, Kathryn ;
Church, Heather ;
Merrigan, Christine ;
Hughes, Joanne ;
Evans, Pamela ;
Campbell, Helen ;
Bonney, Denise ;
Newman, William G. ;
Bigger, Brian W. ;
Broomfield, Alexander ;
Jones, Simon A. ;
Wynn, Robert F. .
ORPHANET JOURNAL OF RARE DISEASES, 2021, 16 (01)