Grant: Planning and Dissemination Grant - Institute Community Support

Leukodystrophies are a group of rare disorders affecting the white matter (myelin) of the brain. Myelin serves as insulation and protection for nerve cells, allowing for normal conduction of nerve impulses. Leukodystrophies are divided in two categories, namely hypomyelinating, i.e. lack of myelin deposition during development, and non-hypomyelinating, i.e. unhealthy myelin, based on brain scan characteristics. The vast majority of leukodystrophies are progressive diseases leading to worsening disabilities and early death. With the advances in genetic research of the recent years, new treatments have been discovered and are being tested for certain subtypes of leukodystrophies. In clinic, families of affected patients always ask questions about those novel therapies. To cater to these families, we have decided that the objective of this Canadian Conference will be to educate families about the current and novel therapies currently available and in development for the leukodystrophies. Patients will also be offered to participate in our multidisciplinary clinic and to participate in our research project on the leukodystrophies.