Crystal HeadshotInvestigator: Dr. Ron Crystal

Disease: Batten Disease

Research Description: Batten Diseases comprise a group of genetic diseases that affect children. Children with these gene defects are missing key enzymes that help to break down waste products in brain cells. These waste products build up, and eventually destroy the brain cells. This causes a loss of function in these children, and eventually death. Dr. Crystal and his team are undertaking a pilot clinical trial to determine the impact of putting a working copy of the affected gene into a virus that can then be injected into the brain. These brain cells would produce the missing enzyme, allowing the brain cells to survive and the children to thrive. If successful, this study could produce a life-saving treatment for children with Batten Disease.

CWR funding role: Primary funder

CWR Start date: November 2010

Sign up for our eNewsletter today!


First Name

Cures Within Reach


All funds donated to Cures Within Reach may be tax deductible for federal income tax purposes.  CWR is recognized by the IRS as a 501(c)3 tax-exempt not-for-profit organization, tax ID 20-3620169.


Monday the 25th. Copyright 2018 Cures Within Reach.