The patient has a genetic disorder known as mucopolysaccharidosis type I, or MPS I. His body doesn't produce an essential enzyme that helps to break down long-chain sugars inside cells. Without this enzyme, these sugars build up in the eyes, heart, bones, and elsewhere.
Sean Ainsworth, CEO of Immusoft, says the initial patient is doing well after receiving the experimental therapy in mid-November. "So far, so good," he says.
[
add
]
[
|
|
...
]