News Headlines Article

Marin doctor seeks cures for rare childhood diseases
The Mercury News

Only one out of every 100,000 people on Earth is born with mucopolysaccharidosis I, or MPS-I, a genetic disorder that can cause organ damage in affected children. It’s a much rarer condition than birth disorders such as cystic fibrosis, which affects about one out of 2,000 children, or Down syndrome, which affects about one in 900. As a result, it’s much more difficult for researchers to find funding for studies of drugs that could help or even cure children with these conditions — or to locate test subjects for those studies.