Leukodystrophies

According to the European Leukodystrophies Association, "leukodystrophies, which refers to a group of rare genetic diseases, destroy the central nervous system (brain and spinal cord) in children and adults. They affect the myelin, the white substance coating the nerves like an insulating sheath." Please find more information on the ELA's website.

We provide the list of leukodystrophies centres, members of ERN-RND, as well as the contact details of medical experts.

All leukodystrophies ERN-RND centres

• ERN-RND approved diagnostic flowcharts
  Diagnostic flowcharts for leukodystrophies

• ERN-RND recommended disease rating scales
• ERN-RND endorsed guidelines

• Clinical trials at ERN-RND centres
• International clinical studies & trials
  ClinicalTrials.gov

• Registries to which ERN-RND members contribute
  Metachromatic leukodystrophy
  - Leukonet
  Leukodystophies
  - LeukoTreat
• Other registries

Patient organisations are non-profit organisations which are patient-focused where patients and/or carers represent a majority of members in governing bodies (European Medicines Agency, 2014). Herewith, we provide a list of national and European patient organisations that work in the disease area that is covered by ERN-RND.

All patient organisations

The listed study groups and disease networks are external networks and groups, which are leaders in the field of research for these disease groups. ERN-RND’s work is based on and complimentary to the work being done by these networks.

• Deutsches Leukodystrophie Netzwerk
• Global Leukodystrophy Initiative

All useful links, training resources, evidence and publications

We organise e-case meetings on leukodystrophies for consensus discussions for therapeutic stem cell transplantations.