Therapeutics of X-linked adrenoleukodystrophy

X-linked adrenoleukodystrophy is a devastating genetic disorder affecting the adrenal glands and nervous system. Identification of the gene defect and its biochemical consequences has opened up several approaches to therapy. We outline strategies to restore gene function as well as those aimed toward ameliorating its downstream metabolic ramifications. In the coming years the implementation of newborn screening will facilitate the assessment of new therapeutics in the early symptomatic stages of disease.

Section editors:Frederick Kaplan – The University of Pennsylvania School of Medicine, Department of Orthopaedic Surgery, Philadelphia, PA 19104, USAAmber Salzman – Cardiokine, Inc., Philadelphia, PA 19102, USA