PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for patients with rare disease

Joined May 2009
755 Photos and videos
Our 2025 Corporate Social Responsibility Report highlights a year of focus, execution and measurable progress delivering differentiated medicines for people with rare disorders. See how we're driving impact across access, innovation, people & our planet: ow.ly/gwTR50Z6RRt
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On #ClinicalTrialsDay, we honor patients, families, and clinical teams advancing rare disease research. This year's #ResearchRising theme reminds us that progress comes from partnership and trust. Learn more: ow.ly/wJiY50Z1Ml5
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May is Friedreich’s ataxia (FA) Awareness Month and we're proud to honor the strength of those living with this rare condition. Kailey’s journey reflects the challenges many face, from recognizing symptoms to finding support. Learn more: ow.ly/KkRx50YZAmQ
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Some days, the hardest part of living with a rare disorder isn’t what others can see—it’s the uncertainty: navigating genetic testing barriers and painful life changes. During #HDAwarenessMonth, PTC is proud to share stories like April's. Learn more: ow.ly/F1os50YWhRo
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Today, we announced our Q1 2026 financial results, highlighting record performance and continued momentum across the business—led by the global launch of our treatment for #PKU. See the release: ow.ly/lbyh50YWi0T and webcast recording: ow.ly/5l7u50YWi1B
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PTC will participate in three upcoming investor conferences in May and June. Learn more: ow.ly/sGsN50YVbXo
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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
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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
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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
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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
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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
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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.
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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.
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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

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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
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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.

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#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
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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
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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
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