Upstaza 欧盟 - 克罗地亚文 - EMA (European Medicines Agency)

upstaza

ptc therapeutics international limited - eladocagene exuparvovec - metabolizam aminokiselina, urođene pogreške - 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.

Opfolda 欧盟 - 克罗地亚文 - EMA (European Medicines Agency)

opfolda

amicus therapeutics europe limited - miglustat - vrsta ii za skladištenje glikogena - ostali prehrambeni proizvodi i proizvodi metabolizma - opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset pompe disease (acid α- glucosidase [gaa] deficiency).