Each is referred to by the initials of the missing enzyme.
The missing enzyme is like an absentee worker on the assembly line.
After birth, the children were given the missing enzyme in drug form, Dr. Kohn said.
In other cases, infusion therapy is used to replace the missing enzyme.
Another approach that can be taken is enzyme replacement therapy, in which a patient is given an infusion of the missing enzyme.
Research is studying techniques to replace the missing enzyme (arylsulfatase A).
An experiment was then carried out to see whether an injection of the missing enzyme into the hips would help the range of motion and pain.
If all goes as planned, the T-cells will propagate enough of the missing enzyme to help restore the children's immune system to normal or near-normal.
Enzyme replacement therapy adds a working form of the missing enzyme to the body.
The new cells can also provide the missing enzyme for someone with a storage disease.