Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants-Reference-Cited by-同舟云学术

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

Published:2010-01 Issue:1 Volume:99 Page:26-33
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Kishnani Priya S.,Goldenberg Paula C.,DeArmey Stephanie L.,Heller James,Benjamin Danny,Young Sarah,Bali Deeksha,Smith Sue Ann,Li Jennifer S.,Mandel Hanna,Koeberl Dwight,Rosenberg Amy,Chen Y-T

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference42 articles.

1. A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease;Kishnani;J. Pediatr.,2006

2. The natural course of infantile Pompe’s disease: 20 original cases compared with 133 cases from the literature;van den Hout;Pediatrics,2003

3. Frequency of glycogen storage disease type II in The Netherlands: implications for diagnosis and genetic counselling;Ausems;Eur. J. Hum. Genet.,1999

4. Early detection of Pompe Disease by newborn screening is feasible: results from the Taiwan screening program;Chien;Pediatrics,2008

5. Isolation and characterisation of a recombinant, precursor form of lysosomal acid alpha-glucosidase;Fuller;Eur. J. Biochem.,1995

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