Novel therapeutic options for hypophosphatasia

Affiliations

¹ Department of Pediatrics and Adolescent Medicine, Johannes Kepler University Linz, 4020 Linz, Austria.
² Osteology/Clinical Trial Unit, University of Würzburg, 97074 Würzburg, Germany.

Wolfgang Högler et al. J Bone Miner Res. 2025.

. 2025 Apr 21;40(4):451-453.

doi: 10.1093/jbmr/zjaf023.

¹ Department of Pediatrics and Adolescent Medicine, Johannes Kepler University Linz, 4020 Linz, Austria.
² Osteology/Clinical Trial Unit, University of Würzburg, 97074 Würzburg, Germany.

No abstract available

Keywords: Enpp1; Tnsalp; alkaline phosphatase; antisense oligonucleotide; asfotase alfa; hypophosphatasia; osteomalacia; rickets.

Comment in

Preclinical evaluation of the efficacy and safety of adeno-associated virus 8-tissue-nonspecific alkaline phosphatase-D10 in Alpl-/- and AlplPrx1/Prx1 mouse models for the treatment of early and late-onset hypophosphatasia.
de Oliveira FA, Tokuhara CK, Mohamed FF, Narisawa S, Lira Dos Santos EJ, Andras NL, Shadid M, Miyake K, Foster BL, Millán JL. de Oliveira FA, et al. J Bone Miner Res. 2025 Apr 21;40(4):463-477. doi: 10.1093/jbmr/zjaf005. J Bone Miner Res. 2025. PMID: 39799564 Free PMC article.