Grönt ljus för genterapi mot sällsynt muskelsjukdom

FDA har gett klartecken för Novartis nya genterapi Itvisma för behandling av patienter med den sällsynta muskelsjukdomen spinal muskelatrofi (SMA).

By Ella Käck

November 25, 2025 10:59 AM

Behandlingen är godkänd för barn och vuxna från två års ålder med en bekräftad mutation i SMN1-genen (survival motor neuron 1), som orsakar sjukdomen.

– Det här ger patienterna fler behandlingsalternativ, vilket alltid är en positiv utveckling, säger Tracey Dawson, chef för neurovetenskapliga terapier vid Novartis USA, till Reuters.

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