Category: AADC deficiency

  • PTC gets green light for gene therapy Upstaza in EU

    PTC Therapeutic’s Upstaza  gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of the commercial prospects for gene therapies in the bloc. Once full approval is granted Upstaza (eladocagene exuparvovec) will become the first disease-modifying treatment for AADC (aromatic L-amino acid decarboxylase) deficiency, […]