Today we proudly recognize International PKU Day, led by @official_espku to spread awareness and education about living with #PKU. Learn how the dietary challenges of this rare condition can impact the quality of life for both children and adults:

We are proud to announce the European Commission has granted marketing authorization for our medicine to treat children and adults living with phenylketonuria (PKU). Learn more:

Our CEO, Dr. Matthew Klein and CFO, Pierre Gravier will participate in the Goldman Sachs 46th Annual Global Healthcare Conference 2025 today June 10 at 9:20 a.m. ET. Tune in to our fireside chat to hear our latest updates:

Our leaders will participate in the William Blair 45th Annual Growth Stock Conference 2025 today June 3 at 10:20 a.m. ET. Tune in to our fireside chat to hear our latest updates:

Our leaders will be participating in the RBC Capital Markets Global Healthcare Conference 2025 today, May 20 at 2:05 p.m. ET. Tune in to our fireside chat to hear our latest updates:

We invite you to hear from PTC's leaders at the Bank of America Securities 2025 Health Care Conference today, May 13. Tune in to our live webcast at 1 p.m. ET (10 a.m. PT) to learn more about the company's latest updates:

Today we announced our first quarter 2025 earnings, highlighting a promising start thanks to strong revenue performance and a steady progression of global launch activities following the recent positive CHMP opinion for our potential PKU treatment.

Today, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has given a positive opinion on PTC's treatment option Sephience (sepiapterin) for adults and children with phenylketonuria (PKU). Learn more:

PTC's leaders will present at four upcoming investor conferences this May and June. Learn more:

Today we announced that our leaders will report the company's first-quarter 2025 financial results and provide an update on the company's business and outlook on Tuesday, May 6 at 4:30 p.m. ET. Learn more:

We are pioneering innovative treatments for patients around the world and are proud to share that our #genetherapy for AADC deficiency (#AADCd) has received approval in Hong Kong.

Our teams were thrilled to connect with the #Friedreichsataxia (FA) community at the 2025 #MDAConference and share data from our #FA program. We're proud to be developing a potential treatment for children and adults living with this rare disorder.

It's been a productive week for us at #ACMGmtg25. We presented new data from our sepiapterin Phase 3 APHENITY trial and open-label extension study demonstrating the potential to treat children and adults living with #PKU. Learn more:

We're excited to be onsite at this year's #ACMGMtg25 in Los Angeles to to share data on our #PKU and #AADCd programs.

We're honored to be named one of Gallup's 2025 Exceptional Workplace Award winners for Strengths Development for the fourth time! Learn more:

PTC will be participating in the Barclays 27th Annual Global Healthcare Conference today, March 11 at 12:30 p.m. ET. Tune in to our fireside chat to hear our latest updates:

PTC will be participating in the TD Cowen 45th Annual Health Care 2025 Conference today, March 4 at 11:10 a.m. ET. Tune in to our fireside chat to hear our latest updates:

On this #RareDiseaseDay, we celebrate the inspiring rare disease community who motivate us to excel. We’re proud to raise awareness of the 6,000+ identified rare diseases and more than 300 million people worldwide living with a rare condition.

Today, our CEO Matthew B. Klein, M.D. shared an update about PTC’s outstanding year of execution in 2024. From record revenue to key regulatory milestones, we achieved remarkable success across all areas of the business last year. Learn more:

The FDA has accepted our NDA for vatiquinone to treat Friedreich's ataxia. Our application has been granted Priority Review and assigned a PDUFA date of Aug. 19, 2025. This is our 4th NDA submitted and accepted for review within the last year. Learn more: