Eladocagene exuparvovec (Upstaza, PTC Therapeutics) 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'.
The dosage schedule is available in the summary of product characteristics for eladocagene exuparvovec.
The company has a commercial arrangement. This makes eladocagene exuparvovec available to the NHS with a discount. The size of the discount is commercial in confidence. It is the company's responsibility to let relevant NHS organisations know details of the discount.