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.