Funding for this research was provided by:
Alexion Pharmaceuticals (NA)
Received: 4 March 2020
Accepted: 26 July 2020
First Online: 6 August 2020
Ethics approval and consent to participate
: Data from three clinical trials were analyzed: Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia, NCT00744042; Extension Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia, NCT01205152; Open-Label Study of Asfotase Alfa in Infants and Children ≤5 Years of Age With Hypophosphatasia, NCT01176266. Site-specific approvals were granted by appropriate authorities.
: With two exceptions, consent for publication of patients’ data was obtained at their enrollment in the clinical trials program for asfotase alfa. Consent for publication of data for the two other patients, who were not included in the clinical trials of asfotase alfa but are included in the reported case series, was provided separately to RP.
: RP, HS and RY are members of the Advisory Board for Alexion Pharmaceuticals, Inc. RP and HS are members of the Speakers Bureau for Alexion Pharmaceuticals, Inc. RP, HS and MZM participated as investigators in the clinical trial program for asfotase alfa, which was sponsored by Alexion Pharmaceuticals, Inc. RP, HS, RY and MZM have received honoraria from Alexion Pharmaceuticals, Inc. The other authors have no financial relationships relevant to this article to disclose.