Not all leukodystrophies have treatments but this is an evolving area. The treatments that are available are Hematopoietic stem-cell transplantation (HSCT) also known as bone marrow transplant (BMT), gene therapy, and drug therapies. To find out more about these treatment options, check the links below and please consult your specialist doctors.
Bone marrow or Hematopoietic stem cell transplant
There are a few clinical trials for leukodystrophy including, Ionis developing Zilganersen for Alexander Disease, Minoryx studying leriglitazone for Adrenoleukodystrophy (ALD). There is also the gene therapy Skysona from Genetix Biotherapeutics for ALD available in the US for boys without a matched donor for HSCT and Libmeldy from Orchard for Metachromatic leukodystrophy (MLD) that has recently approved by EMA and used in England.
We have produced information describing what clinical trials are, who can take part in them, and how to find out information about current clinical trials. To find out more about research and clinical trials, visit our research page.
Treatments are in progress for some leukodystrophies, to find out more, visit our current research page.
Please speak to your healthcare professionals if you see an available clinical trial so they can discuss the suitability and access of that trial for you.
Using the following links you can find out more about the professionals who may be involved in your care, different services and equipment that may be suitable for you.
The information is not condition specific, and in some cases the information may not be relevant to all conditions.
To find out more, visit our condition management page.
