When Glenn ONeill and his wife, Cara, learned about a gene therapy treatment that could save their daughters life, they started a foundation and set off to raise the $2.3 million it would take to fund the manufacturing and clinical trial costs for the drug. Today, thanks to about 26,000 donors from 70 different countries, theyre just $530,000 away from their goal. Their daughter, 4-year-old Eliza ONeill, suffers from Sanfilippo Syndrome-Type A, a disease that causes children to lack an enzyme necessary for normal cellular function. The disease eventually causes a lethal buildup of a toxic material called heparin sulfate, leading to learning disabilities and behavioral problems. The disease is seen in 1 in 70,000 births, and most children born with with Sanfillipo Syndrome-Type A die by the time they are teenagers. While there is currently no cure or treatment for the disease, researchers at Nationwide Childrens Hospital in Columbus, Ohio have found a potential gene therapy that, in a study, successfully rid mice of heparin sulfate buildup. When Cara spoke to lead researcher Dr. Haiyan Fu, principal investigator at the Center for Gene Therapy at Nationwide, and learned about the potential treatment, the family began raising money to fund … Continue reading →