Contact information, map and directions, contact form, opening hours, services, ratings, photos, videos and announcements from PTC Therapeutics, Pharmaceutical Company, 500 Warren Corporate Center Drive, Warren, NJ.
PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for children and adults living with rare disease.
02/23/2026
PKU is rare—but the strength of the PKU community is extraordinary. For many individuals and families, living with PKU means thoughtful planning around food, regular monitoring of Phe levels, and navigating the day-to-day emotions that can come with managing a lifelong condition.
As we approach Rare Disease Day on February 28, we’re proud to amplify voices from the global community—including people living with the condition, healthcare professionals, and advocacy leaders—sharing what life is really like and why awareness and support make such a meaningful difference.
Watch, listen, and share to help us elevate PKU voices. Hear more of their stories here: https://ow.ly/3mHO50YkmRV
02/16/2026
We’re proud to stand with the global rare disease community—and to reinforce our commitment to individuals and families impacted by AADC deficiency ( ).
Because for ultra-rare conditions like AADCd, the journey to answers can be long. As pediatric neurologist Dr. Marcus Wheeler shares, symptoms can overlap with other metabolic disorders, meaning specialists often need to cast a broad diagnostic net—and awareness can be a barrier.
We’re working to help change that through education, advocacy, and support for the AADC deficiency community—so more patients can be recognized sooner and connected to appropriate care.
When Luke Millington joined PTC's Talent Pipeline Program, he was seeking valuable experience at a global biopharmaceutical company. Instead, he found a profound opportunity to support individuals living with Duchenne muscular dystrophy ( ), a condition PTC has passionately advocated for through years of research and innovation.
As a member of our Patient Advocacy team, Luke leverages his lived experience to assist families in navigating the critical transition from childhood to adult DMD care. His journey exemplifies our commitment to empowering patients and healthcare professionals alike, ensuring that every voice is heard and every need is addressed.
Join us in uplifting the rare community all month long by learning more about Luke’s inspiring story. Together, we can make a meaningful difference in the lives of those affected by rare diseases.
At PTC Therapeutics, our commitment to those with significant unmet medical needs pushes us every day. In recognition of this month, we're sharing stories from a few of the brave individuals living with a rare disorder. Meet Tiffany, a courageous mother of two living with Friedreich's ataxia ( ), a rare neuromuscular disorder impacting the central nervous system and heart. Tiffany's journey highlights the power of community and the hope that comes from connecting with others facing similar challenges. 💪❤️ Join us in celebrating her resilience and learn more about her inspiring story: https://ow.ly/FpMH50Y6nzU
01/28/2026
Pam and Kelsey are helping to raise awareness of phenylketonuria (PKU) by sharing their inspiring story. Read more in the link below.
Pam Kowalczyk struggles with the genetic condition PKU, which prevents the body from processing protein. Years ago, she decided to adopt a child with the same condition. Now they manage it together.
12/03/2025
Today, on PKU Awareness Day, we're honored to share Tracy's story.
Tracy is an astrophysicist and NASA contractor who has lived with classical PKU her entire life. Managing a strict low-protein diet, staying balanced while traveling, and navigating school and career demands have shaped her health journey, resilience, and discipline.
Learn more about how PKU has influenced Tracy's life and why advocacy continues to matter: https://bit.ly/4pQ8vND
11/22/2025
When Sarah Chamberlin's daughter was diagnosed with phenylketonuria (PKU), she channeled her experience into creating meaningful change for others living with this rare condition. Today, as the Founder & Executive Director of flok, Sarah continues to advance support and research for the inherited metabolic disorder community. Learn more about her journey: https://bit.ly/48e2i6P
10/23/2025
Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare condition with complex physical and emotional challenges. This AADC Deficiency Awareness Day, we stand with families navigating AADC deficiency and honor their resilience and commitment to overcoming the challenges of this rare condition that is often misdiagnosed. For young Simone and his family, testing at a local hospital didn’t reveal the cause of his symptoms. It was only after traveling to a specialist children's hospital in Italy that they finally received the diagnosis that would change their journey.
Learn more about Simone's path to diagnosis and how has impacted his family: https://bit.ly/43nCt2w
09/25/2025
On this International Ataxia Awareness Day, we are honored to help raise awareness about Friedreich's ataxia ( ). As the most common hereditary ataxia, we recognize the strength of the FA community and shine a light on the brave stories of those living with this rare condition. Sam is a passionate writer whose life changed when he was diagnosed with FA. Learn more about his story and message for the community: https://bit.ly/3Vvnsrd
09/24/2025
The PTC team is excited to be onsite at the 39th European Society for Phenylketonuria and Allied Disorders Treated as Phenylketonuria (E.S.PKU) Annual Conference to hear from the community and to understand the impact of this condition for those living with PKU and their families. Meet us at our booth. See you there!
09/11/2025
Our CEO Matthew Klein, MD joined our team onsite at the recent National PKU Alliance Regional Community Conference in San Diego to hear from the community. A special shout-out to our PTC team who helped make our onsite activities a success.
09/07/2025
PTC is proud to support World Duchenne Awareness Day 2025 ( ). Today serves as an important reminder of the need to raise awareness about Duchenne muscular dystrophy.
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Everyone has a different definition of progress. For the last 20 years, we’ve measured our progress researching rare disease in moments. Smiling ones and crying ones. Moments spent with our patients’ families and ones with their friends. We know that every step forward comes after several steps backward, because we’ve lived it—whether spending time with families in their homes or with our scientists researching in our labs in central New Jersey.
It can be easy to lose yourself as you progress further. Although we’ve grown, our heart remains in the same place, because we’ve never measured ourselves like larger companies do. Our biggest accomplishment has always been the time we can give to all of our families. Whether it’s hours, days, months, or years, every small moment is a big win.
COMMUNITY GUIDELINES
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