♦ NIH Grant to Support a Multi-Center Initiative to Advance Research, Treatment for Leukodystrophies – Read the full article here
♦ Bluebird bio Presents Updated Data from Phase 2/3 Clinical Study of Lenti-D™ Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) at the 13th European Pediatric Neurology Society (EPNS) Congress – Read the full article here
♦ We have a valuable and most important Post Stroke Qualitative Study Questionnaire for those who have had a CADASIL related Stroke and one for those who have had General related Stroke. This study is being carried out by the ACSN in conjunction with our partner the University of Technology Sydney. Click here for more information.
You are likely entering this site because you have received some very unpleasant news about a family member or a friend or you are seeking some personal knowledge about the group of disorders known as the Leukodystrophies.
We greet you as an organisation that has personal understanding and experience of having had an immediate loved one with a Leukodystrophy.
As you explore this site, it is our intention that you find accurate information that you need; be comforted by the support that other families can offer to you and your family and be encouraged by our globally recognised research programs, resources and family support that we will deliver to you.
We are in partnership with the leading Leukodystrophy scientific, medical and support authorities around the world and we offer you the benefit of this powerful knowledge. In doing so, we also extend to you a comforting hand as you continue your walk with Leukodystrophy.
Leukodystrophy is a disorder determined by the following criteria:
- It is genetic
- It has a progressive nature
- It predominately affects the white matter (myelin) in the central nervous system (CNS) and peripheral nervous system (PNS)
- The myelin fails to develop or is destroyed by the dysfunction of a protein or enzyme
It results in devastating symptoms that lead to a dramatically shortened lifespan.
This website is continually being updated and is in the process of being finished.