Hematopoietic cell transplantation does not prevent myelopathy in X‐linked adrenoleukodystrophy: a retrospective study

Background X‐linked adrenoleukodystrophy (X‐ALD) is a peroxisomal metabolic disorder. Male patients develop adrenocortical insufficiency (80 % before 18 years), a chronic myelopathy (adrenomyeloneuropathy (AMN); all in adulthood), or progressive cerebral demyelination (cerebral ALD; 40 % before 18 years). Cerebral ALD is treated with haematopoetic cell transplantation (HCT). It is unknown if AMN still develops in patients with X‐ALD that...