Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study-Reference-Cited by-同舟云学术

Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study

Published:2016-01-14 Issue:3 Volume:39 Page:383-390
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J Inherit Metab Dis

Author:

van Gelder C. M.,Poelman E.,Plug I.,Hoogeveen-Westerveld M.,van der Beek N. A. M. E.,Reuser A. J. J.,van der Ploeg A. T.

Funder

Netherlands Organisation for Health Research and Development

th Frame Program ‘EUCLYD – a European Consortium for Lysosomal Storage Diseases’ of the European Union

Prinses Beatrix Spierfonds

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

http://link.springer.com/article/10.1007/s10545-015-9912-y/fulltext.html

Reference31 articles.

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2. Bijvoet AG, Van Hirtum H, Kroos MA et al (1999) Human acid alpha-glucosidase from rabbit milk has therapeutic effect in mice with glycogen storage disease type II. Hum Mol Genet 8:2145–2153

3. Case LE, Beckemeyer AA, Kishnani PS (2012) Infantile Pompe disease on ERT: update on clinical presentation, musculoskeletal management, and exercise considerations. Am J Med Genet C: Semin Med Genet 160:69–79

4. Case LE, Bjartmar C, Morgan C et al (2015) Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease. Neuromuscul Disord 25:321–332

5. Chakrapani A, Vellodi A, Robinson P, Jones S, Wraith JE (2010) Treatment of infantile Pompe disease with alglucosidase alpha: the UK experience. J Inherit Metab Dis 33:747–750

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