— Campaign highlights unique stories and experiences of the HPP community to raise awareness —
The Every Day with HPP campaign encourages patients with HPP, caregivers, their loved ones and people interested in supporting the HPP community to share their stories of challenges, hopes and aspirations by submitting a photo of a handwritten sign on which they have completed the sentence "Every Day with HPP, I____." These photos and messages will be displayed on EveryDayWithHPP.com, creating an authentic snapshot of everyday life with HPP. Additional information on HPP, tools, videos and downloadable resources will also be featured on the website.
"At Alexion, we are driven every day by our commitment to develop and
deliver life-transforming therapies for patients worldwide who suffer
from severe and life-threatening diseases, including HPP. Initiatives
like HPP Awareness Week further inspire us in this mission," said
In patients with HPP, defective bone mineralization can lead to destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death.1-5 Patients with HPP often experience symptoms in their bones, joints and muscles, as well as in major organs including the brain, lungs and kidneys.1,6 Because of the progressive nature of HPP, new symptoms can appear at any age and symptoms can worsen over time, causing significant disability.1,4,7-10 The devastating outcomes of HPP include fatal seizures and breathing problems in infants; skeletal bowing, frequent fracture and delays in growth and development in children; and debilitating functional impairment and frequent, non-healing fractures in adults.1,4,5,7,11-16
"The goal of HPP Awareness Week is to spread awareness and education
about HPP and support patients with HPP and their loved ones so they
don't feel isolated and alone," said
To learn more about hypophosphatasia or to participate in the Every Day with HPP campaign, please visit EveryDayWithHPP.com.
About Hypophosphatasia (HPP)
HPP is a genetic, chronic and progressive ultra-rare metabolic disease characterized by defective bone mineralization that can lead to destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death.1-5
HPP is caused by mutations in the gene encoding an enzyme known as tissue non-specific alkaline phosphatase (TNSALP).1-2 The genetic deficiency in HPP can affect people of all ages.1 HPP is traditionally classified by the age of the patient at the onset of symptoms of the disease, and pediatric-onset HPP is defined as manifestation of the first symptom prior to 18 years of age.
HPP can have devastating consequences for patients at any stage of life.1 In a natural history study, infants who had their first symptom of HPP within the first 6 months of life had high mortality, with an overall mortality rate of 73% at 5 years.16 In these patients, mortality is primarily due to respiratory failure.1,5,16 In patients surviving to adolescence and adulthood, long-term clinical sequelae include recurrent and non-healing fractures, debilitating weakness, severe pain and the requirement for ambulatory assistive devices such as wheelchairs, wheeled walkers and canes.1,4
About Alexion
Alexion is a biopharmaceutical company focused on serving patients with severe and rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. Alexion is the global leader in complement inhibition and has developed and markets Soliris® (eculizumab) as a treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. Soliris is currently approved in nearly 50 countries for the treatment of PNH and in nearly 40 countries for the treatment of aHUS. Alexion is evaluating other potential indications for Soliris in additional severe and ultra-rare disorders beyond PNH and aHUS, and is developing other highly innovative biotechnology product candidates across multiple therapeutic areas. This press release and further information about Alexion can be found at www.alexionpharma.com.
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REFERENCES
1
2Whyte MP. Hypophosphatasia: nature's window on alkaline
phosphatase function in humans. In: Bilezikian JP, Raisz LG, Martin TJ,
eds. Principles of Bone Biology. Vol 1. 3rd ed.
3Whyte MP, Greenberg CR, Salman N, et al. Enzyme-replacement therapy in life-threatening hypophosphatasia. N Engl J Med. 2012;366(10):904-913.
4Seshia SS, Derbyshire G, Haworth JC, Hoogstraten J. Myopathy with hypophosphatasia. Arch Dis Child. 1990; 65(1):130-131.
5
6Whyte MP. Hypophosphatasia. 2001:5313-5329.
7Skrinar A, Smith J, Smith S, et al. Burden of illness in
children and adults with hypophosphatasia. Survey results presented at
the
8Balasubramaniam S, Bowling F, Carpenter K, et al. Perinatal hypophosphatasia presenting as neonatal epilepticencephalopathy with abnormal neurotransmitter metabolism secondary to reduced co-factorpyridoxal-5'-phosphate availability. SSIEM and Springer. 2009. 1-9.
9Whyte MP, Murphy W, Fallon M, et al. Adult hypophosphatasia
with chondrocalcinosis and arthropathy.
10Fallon MD, Teitelbaum SL, Weinstein RS, et al.
Hypophosphatasia: clinicopathologic comparison of the infantile,
childhood, and adult forms.
11Mohn A, De Leonibus C, Giorgis T, et al. Hypophosphatasia in a child with widened anterior fontanelle: lessons learned from late diagnosis and incorrect treatment. In: Foundation Acta Paediatrica. 2011; 1-5.
12Beck C, Morbach H, Wirth C, et al. Whole-body MRI in the
childhood form of hypophosphatasia.
13Whyte MP, Greenberg CR, Kishani P, et al. Fracture burden
in children and adults with hypophosphatasia. Poster presented at the
2012 European Society of Calcified Tissues Annual Congress,
14Coe JD, Murphy WA, Whyte MP. Management of femoral
fractures and psuedofractures in adult hypophsphatasia.
15Bliuc D, Nguyen N, Milch V, et al. Mortality risk
associated with low-trauma osteoporotic fracture and subsequent fracture
in men and women. The Journal of the
16Whyte MP, Leung E, Wilcox W, et al. Hypophosphatasia: a
retrospective natural history study of the severe perinatal and
infantile forms. Poster presented at the 2014
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