Treatments
In the past, treatment of hypophosphatasia (HPP) primarily focused on managing the specific symptoms experienced by each patient. A collaborative approach involving various specialists, such as pediatricians, dental specialists, and orthopedic surgeons, was commonly employed to develop a comprehensive treatment plan. This often included the use of non-steroidal anti-inflammatory drugs to alleviate joint pain and vitamin supplements to regulate seizures in children with increased cranial pressure. For adults with a higher susceptibility to fractures, internal fixations were often necessary. Surgeons would insert rods into weakened bones to provide additional support and enhance strength.
However, a significant breakthrough occurred in 2015 when the Food and Drug Administration (FDA) granted approval for enzyme replacement therapy as a treatment for HPP. This marked a significant milestone in the management of the condition.
Enzyme Replacement Therapy: A Promising Treatment for Hereditary Hypophosphatasia (HPP)
Hereditary Hypophosphatasia (HPP) can be a challenging condition to manage, especially during the perinatal, infantile, and childhood stages. However, medical advancements have paved the way for a potential solution known as Enzyme Replacement Therapy (ERT). This innovative treatment approach involves replacing the deficient TNSALP enzyme with a specially engineered glycoprotein in a laboratory setting.
The effectiveness of this therapy has been so remarkable that the United States Food and Drug Administration (FDA) has granted it the breakthrough therapy designation. By mimicking the functions of the natural enzyme, the lab-produced glycoprotein offers promising results in alleviating the symptoms and complications associated with HPP.
Alongside the administration of Enzyme Replacement Therapy, healthcare professionals diligently monitor the patient’s calcium balance. Additionally, they may prescribe supplementary treatments as needed to optimize the management of HPP and ensure the best possible outcomes for patients.