The identification of new prognostic biomarkers to improve care for patients with ALD has become particularly relevant now that newborn screening for ALD is being introduced. Early identification of adrenal insufficiency and/or cerebral disease will improve survival and neurological outcome of individual patients. In the current landscape, however, it is not possible to distinguish those newborns at high risk for cerebral ALD from those at low risk. Ideally, the identification of a newborn or child with ALD should be followed by a prediction of the most likely clinical course for that individual, thereby creating the possibility of tailoring of medical treatment and follow-up to the individual characteristics. By following a large cohort of ALD patients prospectively and systematically we aim to identify biomarkers that are predictive of disease course and lead to personalized medicine.
