Skip to main content

A journey with many milestones

2023 NASDAQ bell ringing


  • Matthew B. Klein, M.D. appointed Chief Executive Officer
  • Statistically significant results from sepiapterin Phase 3 PKU trial
  • PTC celebrates its 25th anniversary and 10 years as a public company
Brain image


  • Upstaza™ (eladocagene exuparvovec) for the treatment of AADC deficiency, the first ever gene therapy directly administered into the brain, was approved in Europe and the UK
Microscope and tubes


  • Evrysdi® (risdiplam) approved in the EU and Japan for patients with spinal muscular atrophy (SMA)
  • Received Gallup’s “Don Clifton Strengths-Based Culture” Award
  • Proof of mechanism established for PTC518 demonstrating broad distribution for Huntington’s disease
  • Expanded partnership with the SMA Foundation to advance drug discovery and development research in regenerative medicine
Lab equipment


  • Expanded clinical pipeline to include a treatment for phenylketonuria (PKU)
  • Evrysdi® (risdiplam) approved by the FDA for patients with spinal muscular atrophy (SMA)
Lab equipment


  • Expanded platform technologies with the acquisition of a ferroptosis & inflammation platform to address diseases of oxidative stress
  • Announced clinical results for its gene therapy treatment for patients with AADC deficiency that demonstrated sustained improvements
  • Announced Real-World Evidence demonstrating Translarna™ (ataluren) slows disease progression in patients with Duchenne muscular dystrophy
Lab equipment


  • Addition of Tegsedi® (inotersen) and Waylivra® (volanesorsen) expands the product portfolio in Latin America
  • Acquired a targeted gene therapy platform targeting rare CNS disorders such as AADC deficiency
Lab equipment


  • Acquired Emflaza® (deflazacort) for the treatment of Duchenne muscular dystrophy: The only drug approved in the US for all patients living with Duchenne
Scientest wearing gloves


  • Translarna™ (ataluren) is approved in the EU: The first drug in the world to be approved for Duchenne muscular dystrophy
NASDAQ ticker


  • Successful IPO in the US public markets (NASDAQ: PTCT)


  • PTC and the SMA Foundation partner with Roche to continue the development of a treatment for spinal muscular atrophy (SMA)
Researcher working at desk


  • Awarded grant from the Wellcome Trust that supported research and development of unesbulin


  • Partnered with the SMA Foundation to discover and develop a treatment for spinal muscular atrophy (SMA)
Microscope lenses


  • Initiated the first clinical trials for PTC124, the compound that would become Translarna™ (ataluren), to target the underlying cause of rare disorders caused by a nonsense mutation
DNA strand


  • PTC was founded with the mission to discover, develop, and commercialize treatments for patients with rare disorders

We'd love to hear from you