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http://abcnews.go.com/Health/family-break-gofundme-record-save-child-rare-disorder/story?id=23963169
http://www.gofundme.com/ElizaONeill
http://abcnews.go.com/blogs/health/2014/04/28/boy-7-makes-heartfelt-plea-for-help-to-fund-sisters-lifesaving-cure/
A little girl needs your help!!!
Eliza has Sanfilippo syndrome type A, a rare genetic disorder that causes a deadly buildup of heparin sulfate in her cells. Soon, Eliza will lose the ability to speak, her parents say. After that, sheāll lose the ability to walk and then sheāll develop seizures.
Most children with Sanfilippo type A donāt live far into their teens.
All varieties of the disease affect one in 70,000 live births, according to the National Institutes of Health, but there is no cure.
Doctors at Nationwide Childrenās Hospital in Columbus, Ohio, have been working on a cure for 16 years and have found a gene therapy that works in mice. They hope to try it on humans early next year, but they need about $2 million to make a clinical trial happen.