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SpringerLink电子期刊(2)
NATURE电子期刊(1)
Elsevier电子期刊(10)
ACS电子期刊(1)
在“
Elsevier电子期刊
”中,
命中:
10
条,耗时:小于0.01 秒
在所有数据库中总计命中:
14
条
1.
A novel approach to characterization and categorization of infusion reactions associated with ERT using adverse physiology related groups
作者:
Christine Haller
;
Sunil Agarwal
;
Emil
Kakkis
刊名:Molecular Genetics and Metabolism
出版年:2016
2.
Recombinant human 尾-glucuronidase enzyme replacement therapy for mucopolysaccharidosis type VII: report of the first patient treated
作者:
Joyce E. Fox
;
Emil
D.
Kakkis
;
William S. Sly
刊名:Molecular Genetics and Metabolism
出版年:February, 2014
3.
Repeated intrathecal injections of recombinant human 4-sulphatase remove dural storage in mature mucopolysaccharidosis VI cats primed with a short-course tolerisation regimen
作者:
Dyane Auclair
;
John Finnie
;
Joleen White
;
Timothy Nielsen
;
Maria Fuller
;
Emil
Kakkis
;
Alphonsus Cheng
;
Charles A. O’
;
Neill
;
John J. Hopwood
关键词:
Mucopolysaccharidosis type VI
;
Lysosomal storage disorders
;
Enzyme replacement therapy
;
Immunotolerance
;
Tolerisation
;
Intrathecal therapy
;
Dura mater
刊名:Molecular Genetics and Metabolism
出版年:2010
4.
A dose-optimization trial of laronidase (Aldurazyme
®
) in patients with mucopolysaccharidosis I
作者:
Roberto Giugliani
;
Veró
;
nica Muñ
;
oz Rojas
;
Ana Maria Martins
;
Eugê
;
nia R. Valadares
;
Joe T.R. Clarke
;
José
;
E.C. Gó
;
es
;
Emil
D.
Kakkis
;
Mary Alice Worden
;
Marisa Sidman
;
Gerald F.
关键词:
Mucopolysaccharidosis (MPS) type I
;
Hurler
;
Hurler–
;
Scheie
;
Scheie
;
Laronidase
;
Clinical trial
;
Dose-optimization
;
Glycosaminoglycans
;
Enzyme replacement therapy
刊名:Molecular Genetics and Metabolism
出版年:2009
5.
72. Transforming the development of treatments for lysosomal storage disorders
作者:
Emil
Kakkis
刊名:Molecular Genetics and Metabolism
出版年:2010
6.
Efficacy of Sapropterin Dihydrochloride in Increasing Phenylalanine Tolerance in Children with Phenylketonuria: A Phase III, Randomized, Double-Blind, Placebo-Controlled Study
作者:
Friedrich K. Trefz
;
Barbara K. Burton
;
Nicola Longo
;
Mercedes Martinez-Pardo Casanova
;
Daniel J. Gruskin
;
Alex Dorenbaum
;
Emil
D.
Kakkis
;
Eric A. Crombez
;
Dorothy K. Grange
;
Paul Harmatz
;
Mark H. Lipson
;
Andrzej Milanowski
;
Linda Marie R
;
olph
;
Jerry Vockley
;
Chester B. Whitley
;
Jon A. Wolff
;
Judith
刊名:The Journal of Pediatrics
出版年:2009
7.
Intrathecal enzyme replacement therapy: Successful treatment of brain disease via the cerebrospinal fluid
作者:
Patricia Dickson
;
Michael McEntee
;
Carole Vogler
;
Steven Le
;
Beth Levy
;
Maryn Peinovich
;
Stephen Hanson
;
Merry Passage
;
Emil
Kakkis
关键词:
Mucopolysaccharidosis I
;
Lysosomal storage disorder
;
Intrathecal
;
Enzyme replacement therapy
;
Central nervous system
;
Cerebrospinal fluid
;
Hurler
;
Scheie
;
Hurler–
;
Scheie
;
Pachymeningitis
刊名:Molecular Genetics and Metabolism
出版年:2007
8.
Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (laronidase)
作者:
James E. Wraith
;
Lorne A. Clarke
;
Michael Beck
;
Edwin H. Kolodny
;
Gregory M. Pastores
;
Joseph Muenzer
;
David M. Rapoport
;
Kenneth I. Berger
;
Stuart J. Swiedler
;
Emil
D.
Kakkis
;
Tanja Braakman
;
Elenie
关键词:
GVHD
;
Graft-versus-host disease
;
HCT
;
Hematopoietic stem cell transplantation
;
SS
;
Standard score
刊名:The Journal of Pediatrics
出版年:2004
9.
Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I
作者:
Kakavanos
;
Revecca
;
Turner
;
Chris T
;
Hopwood
;
John J
;
Kakkis
;
Emil
D
;
Brooks
;
Doug A
刊名:The Lancet
出版年:2003
10.
Long-Term and High-Dose Trials of Enzyme Replacement Therapy in the Canine Model of Mucopolysaccharidosis I
作者:
Kakkis
;
Emil
D.
;
McEntee
;
Michael F.
;
Schmidtchen
;
Artur
;
Neufeld
;
Elizabeth F.
;
Ward
;
Daniel A.
;
et. al.
刊名:Biochemical and Molecular Medicine
出版年:1996
1
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