[GNE myopathy: proven failure of sialic acid supplementation… what's next?]

Med Sci (Paris). 2017 Nov:33 Hors série n°1:55-56. doi: 10.1051/medsci/201733s111. Epub 2017 Nov 15.
[Article in French]
No abstract available

MeSH terms

  • Animals
  • Clinical Trials, Phase II as Topic
  • Disease Models, Animal
  • Distal Myopathies / drug therapy*
  • Distal Myopathies / genetics
  • Distal Myopathies / metabolism
  • Distal Myopathies / pathology
  • Drug Evaluation, Preclinical
  • Humans
  • Mice
  • Mice, Transgenic
  • N-Acetylneuraminic Acid / therapeutic use*
  • Protein Processing, Post-Translational / drug effects
  • Protein Processing, Post-Translational / physiology
  • Research Design / standards
  • Treatment Failure

Substances

  • N-Acetylneuraminic Acid

Supplementary concepts

  • Distal myopathy, Nonaka type