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MF

Mary Frances Harmon

Senior Vice President, Corporate and Patient Relations

PTC Therapeutics

PTC Therapeutics Pipeline

DrugIndicationPhase
Evrysdi (risdiplam)Spinal Muscular Atrophy (SMA)Approved
Translarna (ataluren)Duchenne muscular dystrophy (nmDMD)Approved
Upstaza (eladocagene exuparvovec)Aromatic L-amino acid decarboxylase (AADC) deficiencyApproved
PTC518Huntington's diseasePhase 2
Vatiquinone (PTC743)Friedreich's ataxiaPhase 3
Utreloxastat (PTC857)Amyotrophic Lateral Sclerosis (ALS)Phase 2
PTC923Phenylketonuria (PKU)Phase 3