Adrenoleukodystrophy is a rare inherited disorder identified in patients with a defective gene that produces a protein called ALD; which leads to progressive brain damage. ALD is a transporter required for the break down fats. Defects in ALD lead to the build up of fats and the eventual damage of the myelin sheath that protects nerves. For many years the only treatment for this has been bone marrow transplants, which is limited in the availability of donors and let us not forget the number of complications that could arise.

A new treatment has shown itself in the form of gene therapy. More >