Ny behandling mot kortväxthet kan vara på väg

Ett nytt potentiellt läkemedel mot kortväxthet är föremål för en prioriterad granskning av den amerikanska läkemedelsmyndigheten FDA – men har nu råkat ut för en försening.

By Anders Göransson

November 28, 2025 11:18 AM

Läkemedelskandidaten Transcon CNP (navepegritide) utvecklas av danska Ascendis Pharma för behandling av achondroplasi, som är den vanligaste formen av skelettdysplasi, eller kortväxthet.

Sjukdomen orsakas av en mutation i FGFR3‑genen, som leder till att armar och ben blir kortare än normalt, medan bålen är mer normalt utvecklad.

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