Ayvakyt Európai Unió - magyar - EMA (European Medicines Agency)

ayvakyt

blueprint medicines (netherlands) b.v. - avapritinib - gastrointestinal stromal tumorok - egyéb daganatellenes szerek, a protein kináz inhibitorok - ayvakyt is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (gist) harbouring the platelet-derived growth factor receptor alpha (pdgfra) d842v mutation.

Upstaza Európai Unió - magyar - 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.