Imagine that, despite a baby being born with a rare and often fatal disease, the cure was born previously with the baby’s sibling.
Hurler syndrome is an inherited disorder where a child has a deficiency of a key enzyme needed to break down complex sugars (called glycosminoglycans.) The buildup of sugar results in progressive organ deterioration and ultimately, death in childhood. The symptoms may only appear after age 3 and after the brain has already undergone damage.
According to a study published in the Annals of Neurology
, treatment of Hurler syndrome with umbilical cord blood transplantation before
9 months of age leads to normal cognitive development. Early transplantation also predicted better outcomes for language skills and adaptive behaviors. More specifically, children transplanted at 12-25 months of age functioned cognitively at a level 2 to 5.3 years below that of those transplanted at 4 months.