Effectiveness of asfotase alfa for treatment of adults with hypophosphatasia: results from a global registry.

"Declarations Ethics approval and consent to participateThe Global HPP Registry is sponsored by Alexion, AstraZeneca Rare Disease (Boston, MA, USA) and overseen by a scientific advisory board of clinical experts, including employees of Alexion. The protocol for the registry was approved by the institutional review board (or equivalent) at each participating site and is being conducted in accordance with European Medicines Agency Good Pharmacovigilance Practices, International Society for Pharmacoepidemiology Guidelines for Good Pharmacoepidemiology Practices, and the Declaration of Helsinki. All patients or parent/legal guardians provided written informed consent and approval to release medical records for inclusion in the registry. Consent for publicationNot applicable. Competing interestsPSK, KMD, GAMM, AL, CRG, KO, WH, and LS consult for/have received research funding/honoraria from Alexion, AstraZeneca Rare Disease. AP, AM, and SF are employees of and may own stock/options in Alexion, AstraZeneca Rare Disease. The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. Competing interests PSK, KMD, GAMM, AL, CRG, KO, WH, and LS consult for/have received research funding/honoraria from Alexion, AstraZeneca Rare Disease. AP, AM, and SF are employees of and may own stock/options in Alexion, AstraZeneca Rare Disease. The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest."

"Funding This study was sponsored by Alexion, AstraZeneca Rare Disease (Boston, MA, USA). The Global HPP Registry, also sponsored by Alexion, AstraZeneca Rare Disease, is overseen by a scientific advisory board comprising HPP clinical experts, including employees of Alexion, AstraZeneca Rare Disease. Alexion, AstraZeneca Rare Disease held roles in conception and design of the registry, collection of data, and statistical analyses, as described in the Author Contributions section."