Sanfilippo Syndrome is considered an "orphan disease". That means the disease has not been adopted by the pharmaceutical industry because it provides little financial incentive for the private sector to make and market new medications to treat or prevent it. There are simply too few children diagnosed each year for the pharmaceutical companies to invest the millions of dollars it takes to bring a drug or treatment to market. So, the burden is left to families and their 501c3 charities to raise funds for research and clinical trials.
This $250,000 grant from Pepsi could get us closer to finding an effective treatment. Please VOTE!!