by the abnormal beta-oxidation of very long chain fatty acids (VLCFA). In 35-40% of children
with ALD, an acute inflammatory process occurs in the central nervous system (CNS)
leading to demyelination that is rapidly progressive, debilitating and ultimately fatal.
Allogeneic hematopoietic stem cell transplantation (HSCT) can halt disease progression in
cerebral ALD (C-ALD) if performed early. In contrast, for advanced patients the risk of …