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01-02-2016 | Adis Drug Evaluation

Asfotase Alfa in Perinatal/Infantile-Onset and Juvenile-Onset Hypophosphatasia: A Guide to Its Use in the USA

Author: Lesley J. Scott

Published in: BioDrugs | Issue 1/2016

Abstract

Subcutaneous asfotase alfa (Strensiq™), a first-in-class bone-targeted human recombinant tissue-nonspecific alkaline phosphatase (TNSALP) replacement therapy, is approved in the USA for the treatment of patients with perinatal/infantile- or juvenile-onset hypophosphatasia (HPP). In clinical trials, asfotase alfa was an effective and generally well tolerated treatment for perinatal/infantile- and juvenile onset-HPP through at least 3 and 5 years’ treatment, respectively. Relative to untreated age-matched, juvenile-onset-HPP historical control cohorts, survival and ventilation-free survival were significantly prolonged in asfotase alfa-treated patients, consequent to preceding improvements in bone mineralization.

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Title: Asfotase Alfa in Perinatal/Infantile-Onset and Juvenile-Onset Hypophosphatasia: A Guide to Its Use in the USA
Author: Lesley J. Scott
Publication date: 01-02-2016
Publisher: Springer International Publishing
Published in: BioDrugs / Issue 1/2016
Print ISSN: 1173-8804
Electronic ISSN: 1179-190X
DOI: https://doi.org/10.1007/s40259-016-0161-x

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Asfotase Alfa in Perinatal/Infantile-Onset and Juvenile-Onset Hypophosphatasia: A Guide to Its Use in the USA

Abstract

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

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