Response of 33 UK patients with infantile-onset Pompe disease to enzyme replacement therapy-Reference-Cited by-同舟云学术

Response of 33 UK patients with infantile-onset Pompe disease to enzyme replacement therapy

Published:2015-10-26 Issue:2 Volume:39 Page:261-271
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J Inherit Metab Dis

Author:

Broomfield A.,Fletcher J.,Davison J.,Finnegan N.,Fenton M.,Chikermane A.,Beesley C.,Harvey K.,Cullen E.,Stewart C.,Santra S.,Vijay S.,Champion M.,Abulhoul L.,Grunewald S.,Chakrapani A.,Cleary M. A.,Jones S. A.,Vellodi A.

Publisher

Wiley

Subject

Genetics(clinical),Genetics

Link

http://link.springer.com/content/pdf/10.1007/s10545-015-9898-5.pdf

Reference32 articles.

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2. Banugaria SG, Prater SN, Patel TT et al (2013) Algorithm for the early diagnosis and treatment of patients with cross reactive immunologic material-Negative classic infantile pompe disease: a step towards improving the efficacy of ERT. PLoS One 8:e67052

3. Banugaria SG, Prater SN, Ng YK et al (2011) The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease. Genet Med 13:729–736

4. Baruteau J, Broomfield A, Crook V et al (2014) Sucessful Densitisation in a patient with CRIM-Positive Infantile Onset Pompe disease. JIMD Rep 12:99–102

5. Byrne BJ, Kishnani PS, Case LE et al (2011) Pompe disease: design, methodology, and early findings from the Pompe Registry. Mol Genet Metab 103:1–11

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