Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study - Concepedia

Concepedia

Publication | Open Access

Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study

DOI Full Paper Access

52

Citations

34

References

2021

Year

Nuria Carrillo, May Christine V. Malicdan, Petcharat Leoyklang, Joseph A. Shrader, Galen O. Joe, Christina Slota, John Perreault, John D. Heiss, Bradley Class, Chia‐Ying Liu,

Genetics in Medicine

Abstract

ManNAc showed long-term safety, biochemical efficacy consistent with the intended mechanism of action, and preliminary evidence clinical efficacy in patients with GNE myopathy.

References

	Year	Citations

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