Queen Square Centre for Neuromuscular Diseases


GNE myopathy-Disease Monitoring Programme (GNE-DMP): A registry and prospective observational natural history study to assess HIBM disease

Sponsor / Funder Ultragenyx and Newcastle University
CI Professor Hanns Lochmüller
Sites Newcastle
Contact details becky.davis@newcastle.ac.uk
More information

This is a public-private partnership between Ultragenyx Pharmaceutical Inc. (USA) and Newcastle University (UK).

Primary objective

Tto improve knowledge and treatment development of GNE myopathy. This will be done by collecting data on clinical presentation and progression.

The program allows a combination of longitudinal data collected through:

  • online global patient registry
  • hospital based natural history study

The Natural History study is performed by selected centres in Europe, Middle East and North America.

Following approval from the Steering Committee and Ethical Committee, anonymous data from the registry will be available to:

  • clinicians
  • researchers
  • patients
  • families
  • and patient organisations

This information could provide insight into the disease and help drive clinical trials. That could lead to better treatment strategies.