They’re in a race to save their children with a rare disease. A new drug has given them hope.
Like other children with Hunter syndrome, Cole is deficient in an enzyme required to break down certain molecules. Over time, toxins accumulate, and the genetic disorder ravages children’s organs, including their heart — and in many cases, their brain, leading to dementia-like symptoms. The condition, also called mucopolysaccharidosis type II, or MPS II, affects about...
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