Then, a routine blood draw revealed that Evie had low alkaline phosphatase (ALP), which enabled them to diagnose her with hypophosphatasia (HPP) – an ultra-rare, genetic, potentially life-threatening metabolic condition that prevents minerals such as calcium and phosphate from being properly deposited in bones.1-4
Her parents were told that Evie’s case was very severe and that there were no approved or effective treatment options. At three months old, Evie was enrolled in a clinical trial to receive an investigational targeted enzyme replacement therapy, which was approved in 2015 and is now known as STRENSIQ® (asfotase alfa). Evie continues to receive STRENSIQ today.
Evie is, as her mother says, “writing the book on where we go from here. Her story gives other parents with special needs kids hope that they can be okay, and things can be good.”*
*The results from use of STRENSIQ may vary.