Einmal-Gentherapie zeigt transformativen therapeutischen Nutzen

Neue Studiendaten belegen eine altersgemässe Entwicklung und das Erreichen motorischer Meilensteine bei präsymptomatischen Kindern mit SMA mit Hilfe der Einmal-Gentherapie, ohne jegliche Atem- oder Ernährungsunterstützung oder schwerwiegende, behandlungsbedingte unerwünschte Ereignisse.

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Einmal-Gentherapie zeigt transformativen therapeutischen Nutzen

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