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An experimental therapy helps a little girl with a rare bone condition continue to live her life.
Evie Elsaesser, 4, was expected to survive for only a few moments after her birth. Elsaesser was born with hypophosphatasia, a bone disease that doesn’t allow the skeleton to form correctly while it is growing and is considered life threatening.
According to Good Morning America, the condition keeps an individual’s bones extremely fragile, almost soft. Only 1 in 200,000 people have severe cases of this disease.
Elsaesser was placed in a clinical trial that would give her doses of enzyme to assist the deficiency of it due to hypophosphatasia, Live Science reports. She received regular injections that helped firm up her bones so that they would be less fragile.
While past treatments of this kind have been unsuccessful, this new form of treatment has re-engineered protein that helps the bones in a more efficient manner.
It is hoped that this treatment will eventually replace many of Elsaesser’s other medications.