PTC Therapeutics

09/30/2026

Hello, Fabry community.

For more than 25 years, PTC has worked to bring meaningful therapies to children and adults living with rare diseases: six approved treatments across 50+ countries, including a gene therapy. Now, we're honored to introduce ourselves to the Fabry community as we advance the investigational gene therapy ST-920.

Our Patient Engagement team has loved meeting so many of you at the recent Fabry conference and this is just the beginning. Read our letter to the community for what's ahead, including our planned FDA submission before the end of 2026.

🔗 https://ow.ly/aQ6g50ZTeen

09/25/2026

Today is International Ataxia Awareness Day.

Friedreich's ataxia (FA) is a rare, progressive genetic disease that affects movement, coordination and so much more. But if there's one thing the FA community continues to show us, it's this: no one should face this journey alone.

Today we're honored to share some of the voices of individuals living with FA, whose strength is matched only by the community that surrounds them. Their experiences remind us that connection, understanding and shared purpose can be powerful.

At PTC, we're inspired every day by the resilience of the rare disease community, and we remain committed to standing alongside them.

👉 Read their stories: https://ow.ly/ejWO50ZQXL2

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...