PTC Therapeutics

This #NationalPKUAwarenessMonth, we’re honoring the resilience of the PKU community. Living with PKU is more than a diet—it takes constant planning, advocacy and support. Amanda’s story, alongside her mom Jill, helps make the invisible visible. Learn more: ow.ly/kfKX50YTBSG

On Rare Disease Day, we recognize the millions living with rare conditions. At PTC, we know that progress starts with listening to those impacted. Together, we can make a difference. Learn more: ow.ly/pwXK50YliIh

PTC will participate in three upcoming investor conferences in March, including TD Cowen 46th Annual Health Care Conference, Leerink 2026 Global Healthcare Conference and the Barclays Global Healthcare Conference. Learn more: ow.ly/PEC450YmECC

PKU is rare—but the PKU community’s strength is extraordinary. Living with PKU means daily planning, monitoring, and resilience. This Rare Disease Day, we’re amplifying their voices. Watch, listen, and share their stories: ow.ly/3sAY50Yj1Zg

Today, we announced our Q4 and full-year 2025 financial results, highlighting strong revenue performance that sets the stage for continued growth and innovation in the coming year. Learn more in our release: ow.ly/QStt50Yirls and webcast recording: ow.ly/2aOO50Yiq6Y

Proud to stand with the global #RareDisease community and families impacted by AADC deficiency (#AADCd). As Dr. Marcus Wheeler notes, symptoms can overlap with other disorders—so awareness matters. We’re committed to providing education, support, and earlier recognition.

PTC was honored to receive BioNJ's 2026 Innovator Award for the FDA approval of our treatment for PKU. Thank you, BioNJ, and congrats to everyone advancing the future of medicine.

Sometimes, the right opportunity finds you. Luke Millington turned his internship at PTC into a career supporting the #DMD community. Read his inspiring story: ow.ly/Amcu50YavHM

PTC will host a web conference call to report our fourth-quarter and full-year 2025 financial results and provide an update on the company’s business on Thursday, Feb. 19, 2026, at 4:30 p.m. ET. Learn more: ow.ly/nNs950Y9i0G

At PTC, our commitment to those with significant unmet medical needs drives us every day. In honor of Rare Disease Day this month, we're sharing the story of Tiffany, a mother of two living with #FA, a rare, neuromuscular disorder. Learn more: ow.ly/HZyr50Y6o3M.

Pam and Kelsey are helping to raise awareness of phenylketonuria (PKU) by sharing their inspiring story. Read more: ow.ly/3xa950Y4yxJ

#ICYMI: At the J.P. Morgan 2026 Healthcare Conference, PTC Therapeutics shared key updates on our strong positioning for success in 2026 and beyond. Learn more: ow.ly/UZhU50XXA8h

Today, our CEO Dr. Matthew Klein, shared highlights from a successful 2025 and an outlook for what we expect to be an exciting 2026 at the J.P. Morgan Healthcare Conference. Learn more: ow.ly/Q8gR50XVF8L

Our CEO Matthew Klein, M.D., will present at the annual J.P. Morgan 2026 Healthcare Conference on Monday, Jan. 12 at 9 a.m. PST/12 p.m. EST. Learn more: ow.ly/49qw50XSC9a

#BreakingNews: Japan has approved our treatment for children and adults living with phenylketonuria (#PKU), marking a meaningful step forward for the PKU community. Learn more: ow.ly/5tRT50XN9Me

ICYMI: Our CEO Matthew Klein, MD, participated in the Citi 2025 Global Healthcare Conference yesterday to share insights on how PTC's strong Q3 performance has set the stage for a successful future. Listen for more: ow.ly/6yjj50XC91x

Tracy's PKU journey shows why awareness and support matter. On #PKUAwarenessDay, we're proud to share her story and highlight the daily discipline behind living with this rare disease. Learn more: youtu.be/_XO6w44ca7A

Miss PTC’s R&D Day? Catch up on the latest updates on our science, pipeline progress, and insights in the full replay: bit.ly/48ANwHx

PTC announced that it will host an R&D Day on Dec. 2 in New York City to discuss the company's pipeline. The event will also be webcast. Learn more: ow.ly/OBxY50XvUj1

We're honored that our #genetherapy treatment for #AADCdeficiency received the Therapeutics Innovation of the Year by BioTech Breakthrough Awards, a milestone in advancing care for patients with a rare disease.