Hereditary Inclusion Body Myopathy

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