Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease
Author:
Funder
Valerion Therapeutics
Publisher
Springer Science and Business Media LLC
Subject
Genetics(clinical),Drug Discovery,Molecular Medicine
Link
http://link.springer.com/content/pdf/10.1007/s00109-017-1505-9.pdf
Reference35 articles.
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2. Kishnani PS, Hwu WL, Mandel H, Nicolino M, Yong F, Corzo D (2006) A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease. J Pediatr 148(5):671–676
3. Muller-Felber W, Horvath R, Gempel K, Podskarbi T, Shin Y, Pongratz D, Walter MC, Baethmann M, Schlotter-Weigel B, Lochmuller H et al (2007) Late onset Pompe disease: clinical and neurophysiological spectrum of 38 patients including long-term follow-up in 18 patients. Neuromuscul Disord 17(9–10):698–706
4. Kishnani PS, Beckemeyer AA, Mendelsohn NJ (2012) The new era of Pompe disease: advances in the detection, understanding of the phenotypic spectrum, pathophysiology, and management. Am J Med Genet C Semin Med Genet 160(1):1–7
5. Angelini C, Semplicini C (2012) Enzyme replacement therapy for Pompe disease. Curr Neurol Neurosci Rep 12(1):70–75
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