The goal of the project is to develop the fundamental principles for tests that allow an early identification of the cerebral form of the X-ALD disease and are suitable for monitoring relevant therapies. X-ALD is an inheritable metabolic disorder that damages the central nervous system and leads to the early death of patients.
X-Linked Adrenoleucodystrophy (X-ALD) can either result in cerebral inflammation and demyelination (cALD) causing the early death of patients or in a degenerative disease of the spinal cord (AMN) causing failure of the motor system. Bone marrow transplantations can positively affect the course of the disease, but are risky and can only be considered for a limited number of patients. In order to develop the tests described above, the molecular mechanism that causes the inflammatory reaction has to be characterised.