Burden of disease in pediatric patients with hypophosphatasia: results from the HPP Impact Patient Survey and the HPP Outcomes Study Telephone interview

Burden of disease in pediatric patients with hypophosphatasia: results from the HPP Impact Patient Survey and the HPP Outcomes Study Telephone interview

Abstract Background Hypophosphatasia (HPP) is a rare, inherited, metabolic bone disease caused by deficient tissue-non-specific isoenzyme of alkaline phosphatase activity that manifests as a broad range of signs/symptoms, including bone mineralization defects and systemic complications. The burden o...

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Bibliographic Details
Main Authors: Eric T. Rush, Scott Moseley, Anna Petryk
Format: Article
Language:English
Published: BMC 2019-08-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Hypophosphatasia
Symptoms
Health-related quality of life
Disease burden
Survey
Online Access:http://link.springer.com/article/10.1186/s13023-019-1167-5

Internet

http://link.springer.com/article/10.1186/s13023-019-1167-5

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