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Upstaza
eladocagene exuparvovec
Aminosyremetabolisme, medfødte fejl
✅ Autoriseret
Avanceret terapi
✅
Ingen aktive alarmer
Denne medicin har ingen igangværende mangel, sikkerhedsbreve eller tilbagekaldelser.
Hvad det bruges til
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 5.1).
⚠️ Patient sikkerhed
No
Officiel EMA-side
https://www.ema.europa.eu/en/medicines/human/EPAR/upstaza