Adrenoleukodystrophy (ALD) is a rare metabolic disease that leads to progressive loss of key cognitive, visual, and motor functions, ultimately resulting in a vegetative state.
The disease prevents the proper metabolism of very long-chain fatty acids (VLCFA) due to the absence of the ALDP protein. As a result, there is an accumulation of these fatty acids in tissues, particularly in the brain, leading to damage to the cells of the central nervous system (CNS) and the adrenal glands.