A Japanese single-center experience of the efficacy and safety of asfotase alfa in pediatric-onset hypophosphatasia.

Yohei SugiyamaTaijiro WatanabeMakiko TajikaTetsuro MatsuhashiMasaru ShimuraTakuya FushimiKeiko IchimotoAyako MatsunagaTomohiro EbiharaTomoko TsuruokaTomoyuki AkiyamaKei Murayama

Published in: Orphanet journal of rare diseases (2022)

AA may be effective in patients with mild to severe pediatric-onset forms of HPP.

Keyphrases

replacement therapy
childhood cancer