Living With HPP

Besides the skeletal manifestations of the disorder, those living with HPP can face other medical, psychological and social challenges in living with the disease.

Besides the skeletal manifestations of the disorder, those living with HPP can face other medical, psychological and social challenges in living with the disease.

For more information, please see our publications that have been medically validated by the Soft Bones Hypophosphatasia Scientific Advisory Board.

Treatment

NEW In the summer of 2015, the first-ever treatment for HPP was approved for use in Japan, Canada and the European Union (EU). Strensiq (asfotase alfa) is indicated for treatment of patients in Japan with HPP, as enzyme replacement therapy for patients with a confirmed diagnosis of pediatric-onset HPP in Canada and for long-term enzyme replacement therapy in patients with pediatric-onset hypophosphatasia HPP to treat the bone manifestations of the disease in the EU. The European approval applies to all 28 EU member states as well as Iceland, Norway, and Lichtenstein. The most common adverse reactions observed in clinical studies were injection site reactions and injection-associated adverse reactions. Most of these reactions were non-serious and mild to moderate in intensity. As always, patients should consult with their healthcare professionals about the treatment options that are right for them. Alexion, the makers of Strensiq, stated that the product will be available in Japan by late Q3 2015, in Germany in October and noted they are expecting regulatory decisions in the U.S. in the second half of 2015.

There continues to be research to learn more about HPP and to develop additional treatments for the disorder. Visit clinicaltrials.gov for more information and to learn about opportunities for HPP patients to participate in ongoing research.

Newly Diagnosed

It can be overwhelming when you or a loved one is first diagnosed with an illness, especially one that can be life-threatening. Because HPP affects every person differently, there is a wide spectrum of symptoms and severity of the condition. It is important to understand the type of HPP and the challenges associated with each. For more information about HPP, check out What Is HPP?

For the more severe forms of the disease, it is recommended to consult a large medical institution or a physician who has clinical experience in treating HPP (more information). It is also important to have a primary care physician who becomes knowledgeable about HPP and can help coordinate care as multiple specialties may be needed to deal with the complications of the disorder, including an orthopedic surgeon, rehabilitation specialist, pain management specialist, obstetrician, medical geneticist, endocrinologist and dentist.

o We have an incredible community of people and families living with HPP. Our Soft Bones community is active on Facebook, Twitter (@softbonesHPP) and RareConnect that allows families and patients to receive the latest updates and connect with others who have and are affected by HPP. While each person’s HPP experience is unique, our mission is to connect, empower and educate those impacted.