Adrenoleukodystrophy and Adrenomyeloneuropathy

The Pediatric Neuro-Immunology Clinic’s Clinical Care and Research Program for ALD and AMN is a special program that offers clinical trials, often available through the Van Haren Lab, for the treatment of adrenoleukodystrophy (ALD) and adrenomyeloneuropathy (AMN). Our clinical team includes specialists in neurology, stem cell transplant, endocrinology and genetics.

X-linked ALD occurs when myelin, or the fatty protective substance that surrounds nerve cells, breaks down. It is the result of a mutation of a specific gene, ABCD1. Children with ALD often begin life healthy but can develop adrenal failure and brain inflammation over their lifetime. Early monitoring and treatment for ALD is essential, as many symptoms are treatable if caught early.

Later in life as adults, children with the ALD gene mutation can develop AMN, which results in problems with walking and bowel and bladder function. Adult patients with AMN are seen and evaluated by doctors at Stanford Health Care.