Resources to Learn More About ALD
Adrenoleukodystrophy (ALD) is a devastating genetic disorder with no cure, primarily affecting the nervous system and adrenal glands. Caused by mutations in the ABCD1 gene, it leads to a toxic buildup of very long-chain fatty acids (VLCFAs) that destroy the protective myelin around nerve cells. The most severe form, childhood cerebral ALD, progresses rapidly and can be fatal without timely intervention. Early diagnosis through newborn screening or genetic testing is critical for accessing treatments that can slow progression, such as stem cell transplants. Raising awareness and supporting research are essential to improving outcomes for those affected.