ManNAc in GNE myopathy: new positive results

A first phase I clinical trial of ManNAc (a precursor of sialic acid) in 22 people with GNE myopathy followed for 8 months, showed the safety of use of the product and an increase of sialic acid in the blood ). 

A new phase II trial in 12 people with GNE myopathy followed for 6 to 30 months has shown: 

  • an increase in sialic acid in the blood and sialylation in the membrane of muscle cells after 3 months, 
  • a slowing of the decline in muscle strength measured in the upper and lower limbs,
  • a decrease in the course of the disease assessed by the GNE-DPM score after 12 months
  • and then after 18 months of treatment, no serious side effects for up to 2.5 years of treatment.

A new phase II clinical trial, this time against placebo, is planned. 

 

Safety and efficacy of N-acetylmannosamNEine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study. Carrillo N, Malicdan M.C, Leoyklang P et al. Genet Med (2021)