PTC Therapeutics

09/07/2026

September 7 marks World Duchenne Awareness Day ( ). Today is an opportunity to recognize the clinicians, researchers and advocacy organizations advancing care for people living with Duchenne muscular dystrophy (DMD).

DMD remains one of the most common hereditary neuromuscular disease diagnosed in childhood. Progress against it depends on close collaboration across the community, from expert care centers and treating clinicians to the patient organizations that drive awareness, education and access.

Today, PTC thanks the healthcare professionals delivering multidisciplinary Duchenne care, and the advocacy partners whose leadership continues to shape research priorities, standards of care and the lived experience of Duchenne families worldwide.

08/28/2026

The Annual Symposium of the Society for the Study of Inborn Errors of Metabolism is more than a meeting. It's a moment for the global metabolic community to come together around a shared purpose.

Karyn Koladicz, PTC's VP of Medical Affairs, Americas, is at to help lead our engagement with clinicians, researchers and advocates working to advance care in PKU (phenylketonuria) and other inherited metabolic disorders.

We're excited to engage in conversations and share data of the breadth and depth of PTC’s work in phenylketonuria (PKU). Each presentation reflects our commitment to advancing science, and just as importantly, to listening to and learning from the patients, families and communities at the heart of our efforts.

That perspective continues to shape our work, sharpen our ambition and challenge us to keep pushing for meaningful progress for the PKU community.

Visiting SSIEM 2026? Stop by the PTC booth to connect with Karyn and our team.

08/27/2026

Scientific progress in PKU (phenylketonuria) doesn't happen in isolation. It's fueled by unmet need and powered by the PKU community.

🌍 is in full swing and Peter Blomqvist, PTC's Head of Medical Affairs in Europe, is on the ground this week connecting with the people driving this field forward.

Progress in PKU is a shared effort:
🧑‍🤝‍🧑 Patients and caregivers move research forward by sharing lived experience and helping define the outcomes that matter most in daily life.
📣 Advocacy organizations raise awareness, connect families to resources, and align priorities across stakeholders.
🩺 Clinicians & researchers advance care, generate data, and deepen how PKU is understood and managed over time.
💊 Pharma brings the resources and science to discover new pathways and deliver safe, effective treatments to patients.

At , Peter is looking forward to discussing more.

📍 Visit the PTC booth to learn more.

08/26/2026

✨ We're at , the Annual Symposium of the Society for the Study of Inborn Errors of Metabolism.

This week, PTC joins the global inherited metabolic disease community with one shared purpose: advancing science to improve the lives of people living with rare conditions like PKU (phenylketonuria).

🧬 Our leadership is on the ground, connecting with the clinicians, researchers and advocates driving this field forward — including Kristina Kempf, PTC's Country Head for Germany and Austria.

Grateful to Kristina and the entire PTC team representing us this week in Finland, along with every clinician, researcher, patient and caregiver partnering with us to change the course of rare disease.

08/14/2026

The PTC team connected with the community in many meaningful ways over the past several months - at community events, through our Navigating PKU programs and in partnership with advocacy groups. Every conversation has been a valuable reminder of the strength and resilience of the PKU community. We remain committed to listening, learning and working alongside the community as we continue our efforts to support people living with PKU and their families. Thank you to everyone who spent time with us! We’re proud to be part of these conversations and inspired by the community every step of the way.

08/11/2026

Managing a like often involves many moving pieces.

PTC Cares connects people in the U.S. living with PKU who are prescribed a PTC therapy and their caregivers with dedicated case managers. PTC Cares helps to coordinate care and provide guidance, so you feel informed and supported.

For any questions or to learn more, reach out to the team at 1-844-4PTC-CARES (1-844-478-2227), available Monday through Friday from 8 a.m. to 6 p.m. ET. or visit the PTC Cares website: https://ow.ly/LUH150ZyHsI

07/15/2026

We’re headed to the NPKUA Community Conference this week and can’t wait to connect with the PKU community. We’ll be there to listen, learn and share resources through meaningful conversations with individuals and families living with PKU. If you’re attending, stop by our booth and meet the PTC team. We look forward to seeing you there! Learn more: https://ow.ly/EoHK50Znwmh

07/08/2026

The Friedreich's ataxia (FA) community is driven by resilience and an unwavering commitment to progress, and PTC is honored to be part of that journey. Our CEO, Dr. Matthew Klein, recently joined Jen Farmer, CEO of the Friedreich's Ataxia Research Alliance (FARA), for an open conversation about vatiquinone, what we've learned from the MOVE-FA study and details on our upcoming PROVE-FA study, which will be enrolling in the coming months.

To everyone in the FA community who has shared your time, your stories, and your trust in research over the years, thank you.

🎥 Watch the full conversation for more:

FARA's Mission is to marshal and focus the resources and relationsh...

06/28/2026

For the phenylketonuria (PKU) community, every day involves choices most people never see, from careful planning and lifelong dietary limits to a quiet cognitive and emotional load that rarely gets spoken aloud.

This International PKU Day (June 28), our global leaders at PTC are putting words to action with their personal Pledge for PKU – a promise to keep listening, keep learning, and keep showing up for the individuals, families, and clinicians who navigate this condition every day.

Join us in reaffirming what this community deserves: hope, access and a future without limits.

🔗 https://ow.ly/mMaL50ZhKTg

06/16/2026

Ahead of International PKU Day at the end of June, we're making space for a conversation that too often stays in the shadows: mental health.

Living with phenylketonuria ( ) is about more than a strict diet. It's the hidden burden of constant planning, the isolation at a dinner table, the emotional toll of a lifelong condition.

In support of E.S.PKU's "Traces of PKU" campaign, we're honored to share the stories of Kevin, Signe and Regina, three voices from the global PKU community who are opening up about stigma, anxiety, resilience and what it really means to live with a rare disease every day.

Their words are a reminder of why PTC remains deeply committed to the PKU community – not just through science – but by listening, amplifying and standing alongside the people we serve.

👉 Read their full stories to learn more: https://ow.ly/P1yu50ZbgIH