Leading the worldwide fight to treat and cure
Tay-Sachs, Canavan, GM1 and Sandhoff diseases

Vayle Rose Fund

In March 2019, just before turning 6 months old, my daughter Vayle Rose was diagnosed with Canavan disease, an extremely rare neurological disorder that causes progressive damage to nerve cells in the brain.  As a single mom whose only child has a life-threatening disease, my life turned completely upside down.  The hopes and dreams I had for her, for us, were shattered and my life felt like it was over.  Since her diagnosis, I have been striving to give Vayle the best life possible as she fights every single day against this horrible disease. 

The goal of Vayle's fund is to underwrite Canavan research, family support, and carrier screening.

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Thank you from the bottom of our hearts for your support!



What is Canavan Disease?

Canavan is a progressive neurological genetic disorder caused by the absence of a vital enzyme known as aspartoacylase (ASPA). ASPA breaks N-acetylaspartate acid (NAA) into building blocks essential for building myelin. Myelin is a fatty membrane (also known as white matter) that forms a proactive coating around each nerve ensuring the nerve functions properly.  It is one of over 50 genetically inherited disorders known as Leukodystrophies Diseases.

What about research?

There are currently two gene therapy programs in development - Aspa Therapeutics licensed Dr. Guangping Gao's Canavan gene therapy work from the University of Massachusetts Medical School in Worcester, Massachusetts. They are currently working on a natural history study to have an even deeper understanding of the progression of Canavan; which will lead to establishing better endpoints in the upcoming clinical trial. The other program is at Rowan University under the purview of Paola Leone, PhD where she is exploring gene therapy using a different approach. More information is pending.

Vayle Rose Fund

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