Adrenoleukodystrophy (ALD) is a serious genetic disorder characterized by the progressive loss of the protein coat (myelin sheath), which protects nerve fibers. Without this protection, nerve cells are more fragile and easily damaged while also having difficulty transmitting signals to and from the brain. The disease also affects the adrenal glands, preventing them from producing enough steroid hormones.
Childhood cerebral adrenoleukodystrophy
Childhood cerebral adrenoleukodystrophy (CALD) is the most severe form of ALD. Symptoms generally begin between the ages of 4 and 10. Early diagnosis is key to treating the disease. However, not all patients can receive a stem cell transplant, the only treatment so far that can halt disease progression.
Without treatment, patients fall into a vegetative state in which they may be awake but not responsive or aware of their surroundings one to two years after the development of neurological symptoms. They may remain alive in this state for up to 10 years before succumbing to the disease.
The progression of adrenomyeloneuropathy (AMN) is generally milder than that of CALD. The symptoms are also milder, but lifespan can still be affected by the disease.
Patients with AMN can be further divided into those with and without cerebral involvement. Much like children with the disease, adults with cerebral AMN have a more severe form of the disease, which can drastically reduce their lifespan once neurological symptoms develop.
Patients without cerebral involvement may have a normal or nearly normal lifespan.
Patients with Addison’s disease have problems with secretions from their adrenal glands — they do not secrete enough of certain hormones.
Some patients, particularly the younger ones, may develop adrenal failure, infection, or kidney failure, all of which can be fatal. Apart from these potential complications, the life expectancy of patients with Addison’s disease is about 10 years shorter than normal life expectancy.
Adrenoleukodystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.