Hereditary inclusion body myopathy (HIBM) is a severe progressive myopathy affecting a weakness in lower limb distal muscles, generally during early adulthood, leading to a loss of muscular strength and function and, in the long run, to the loss of walking ability. The virulence of the disease is progressive and varies from a duration of 10 to 20 years or more.
ClinBio-GNE study
- Reference laboratory: Institute of Myology
ClinBio-GNE’s aim is to study clinical and biological endpoints for hereditary inclusion body myopathies due to the mutation of gene GNE.
HIBM-PMP study
- Reference laboratory: Institute of Myology
This registry and prospective observational natural history study aims at assessing GNE myopathy or hereditary inclusion body myopathy (HIBM).
For further information: HIBM-PMP study webpage
HIBM Phase III trial — UX001-CL301
- Reference laboratory: Institute of Myology
This phase III randomized, double-blind, placebo-controlled study aims at evaluating the efficacy and safety of Sialic acid extended-release tablets in patients with GNE myopathy or hereditary inclusion body myopathy (HIBM).
For further information: HIBM Phase III trial webpage
