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Diagnostik og behandling af hypofosfatasi
Engelsk titel: Diagnostics and treatment of hypophosphatasia Läs online Författare: Hepp, Nicola ; Frederiksen, Anja Lisbeth ; Khosravi, Jalda ; Beck Jensen, Jens-Erik Språk: Dan Antal referenser: 29 Dokumenttyp: Översikt UI-nummer: 19090047

Tidskrift

Ugeskrift for Laeger 2019;181(10)967-70 ISSN 0041-5782 E-ISSN 1603-6824 KIBs bestånd av denna tidskrift Denna tidskrift är expertgranskad (Peer-Reviewed)

Sammanfattning

Hypophosphatasia (HPP) is a rare inborn, metabolic bone disorder caused by mutations in the tissue-nonspecific alkaline phosphatase-encoding gene: ALPL. The diagnosis is based on biochemical, clinical and genetic evaluation. Low levels of ALP is a hallmark in diagnosing HPP. Mild forms may present unspecific symptoms and be more frequent than previously assumed. Adults with HPP may present with low bone mass, however, bisphosphonates are contra­indicated for these patients. Finally, enzyme replacement therapy has opened new therapeutic perspectives regarding severe HPP.