Luxturna Eiropas Savienība - ungāru - EMA (European Medicines Agency)

luxturna

novartis europharm limited  - voretigene neparvovec - leber congenital amaurosis; retinitis pigmentosa - other ophthalmologicals - luxturna kezelésére javallt, a felnőtt, gyermek betegek látás elvesztése miatt öröklött retina dystrophia által okozott megerősítette, biallelic rpe65 mutációk, akik elegendő életképes retinális sejtek.

Roctavian Eiropas Savienība - ungāru - EMA (European Medicines Agency)

roctavian

biomarin international limited - valoctocogene roxaparvovec - antihaemorrhagiás - treatment of severe haemophilia a (congenital factor viii deficiency) in adult patients without a history of factor viii inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (aav5).

Hemgenix Eiropas Savienība - ungāru - EMA (European Medicines Agency)

hemgenix

csl behring gmbh - etranacogene dezaparvovec - b hemofília - other hematological agents - treatment of severe and moderately severe haemophilia b (congenital factor ix deficiency) in adult patients without a history of factor ix inhibitors.