Clinical trial NCT01634750 - References

GNE Myopathy — Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Citation(s)

•   Amir SM, Barker SA, Butt WR, Crooke AC, Davies AG
  Administration of N-acetyl-D-mannosamine to mammals. Nature. 1966 Aug 27;211(5052):976-7.
•   Argov Z, Mitrani-Rosenbaum S
  The hereditary inclusion body myopathy enigma and its future therapy. Neurotherapeutics. 2008 Oct;5(4):633-7. doi: 10.1016/j.nurt.2008.07.004. Review.
•   Bork K, Reutter W, Gerardy-Schahn R, Horstkorte R
  The intracellular concentration of sialic acid regulates the polysialylation of the neural cell adhesion molecule. FEBS Lett. 2005 Sep 12;579(22):5079-83.

A Phase 1 Randomized, Placebo-Controlled, Double-Blind, Escalating Single-Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ManNAc in Subjects With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Details for clinical trial NCT01634750