PTC now seeking US approval of one-time gene therapy Upstaza
PTC Therapeutics has submitted its application seeking the approval of Upstaza (eladocagene exuparvovec) — a one-time gene therapy to correct the underlying cause of aromatic L-amino acid decarboxylase (AADC) deficiency — to the U.S. Food and Drug Administration (FDA). “We are excited to bring Upstaza one step closer to children…