Thelansis

Cerebral adrenoleukodystrophy (CALD) is the most severe manifestation of adrenoleukodystrophy (ALD), a rare, X-linked metabolic disorder. Approximately 40% to 72% of boys diagnosed with ALD will progress to cerebral ALD, typically between the ages of 3 and 12. Cerebral ALD is characterized by a rapidly progressive neurologic decline leading to a severe loss of neurologic function and death in most untreated patients. It is a devastating condition for affected boys and their families, with a profound symptom burden and rapid decline in neurologic function without treatment. Cerebral ALD is associated with six major functional disabilities; they are of particular clinical importance because they can significantly compromise a patient’s ability to function independently. The six MFDs are loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, and complete loss of voluntary movement. It is important to put together a care team as early as possi...