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PTC Therapeutic’s Upstaza has been recommended by both the EMA and CHMP

Both the European Medicines Agency (EMA) and the Committee for Medicinal Products for Human Use (CHMP) has recommended PTC Therapeutic’s gene therapy, Upstaza (eladocagene exuparvovec), for patients 18 months and older with aromatic L-amino acid decarboxylase (AADC) deficiency.