Upstaza Европейски съюз - унгарски - EMA (European Medicines Agency)

upstaza

ptc therapeutics international limited - eladocagene exuparvovec - az aminosav metabolizmus, az inborn hibák - enzymes, other alimentary tract and metabolism products - upstaza is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic l amino acid decarboxylase (aadc) deficiency with a severe phenotype (see section 5.

Roctavian Европейски съюз - унгарски - 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).