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Therapeutic Benefit of Autophagy Modulation in Pompe Disease

  • Published:2018-07 Issue:7 Volume:26 Page:1783-1796
  • ISSN:1525-0016
  • Container-title:Molecular Therapy
  • language:en
  • Short-container-title:Molecular Therapy

Author:

Lim Jeong-A,Sun Baodong,Puertollano Rosa,Raben Nina

Funder

NIH

Genzyme Corporation

Acid Maltase Deficiency Association

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference106 articles.

1. Neural deficits contribute to respiratory insufficiency in Pompe disease;DeRuisseau;Proc. Natl. Acad. Sci. USA,2009

2. Non-muscle involvement in late-onset glycogenosis II;Filosto;Acta Myol.,2013

3. The emerging phenotype of late-onset Pompe disease: A systematic literature review;Chan;Mol. Genet. Metab.,2017

4. Peripheral nerve and neuromuscular junction pathology in Pompe disease;Falk;Hum. Mol. Genet.,2015

5. Neuroimaging findings in infantile Pompe patients treated with enzyme replacement therapy;McIntosh;Mol. Genet. Metab.,2018
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