Canavan is a progressive neurological genetic disorder caused by the absence of aspartoacylase (ASPA), a vital enzyme. ASPA breaks N-acetylaspartate acid (NAA) into building blocks essential for building myelin (“white matter”), a fatty membrane that forms a proactive coating around the nerves to ensure that they function properly.
Canavan is one of over 50 genetically inherited disorders known as Leukodystrophies Diseases. There is currently no treatment for Canavan and it is life-limiting.
For more information, visit the National Tay-Sachs and Allied Diseases Association website.
Note: As science advances, we continue to experience a fast-evolving landscape of potential new therapies. The families portrayed here offer perspective on coping with rare and/or life-limiting conditions. They also each represent a particular moment in time.
If you are considering a clinical trial for your child, please also see the Courageous Parents Network Guided Pathway on Clinical Trials.