What’s in Store for PTC in 2022
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What’s in Store for PTC in 2022
Last week, we presented at the 40th Annual J.P. Morgan Healthcare Conference, an annual healthcare investment symposium connecting healthcare industry leaders, innovators, and the broader healthcare industry. Even though this year’s meeting was (once again) virtual, the excitement for everything that’s to come in 2022 was palpable – especially for us at PTC, as 2022…
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Next Stop: Helping those with PKU
As the expression goes, “the journey is more important than the destination.” For Global Project Leader Neil Smith, PharmD, MBA, both are equally important, as improving the lives of other people is the ultimate destination. Smith has kept this end goal in mind throughout his journey working on PTC923 – an oral therapy in development…
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PTC in 2021: A Year of Expansion
For PTC, 2021 was about expansion – not just literally, as we grew our global reach and added team members, but what we can do for patients, too, through our awareness efforts and continued dedication to scientific innovation. Expansion by definition is the act of becoming larger or more extensive. And in fact, we had so much good news in 2021, it was hard for us to pick the highlights to include…
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Transition to Adult Care in DMD with Dr. Lauren Elman
Dr. Elman is an adult neurologist at the University of Pennsylvania, in this episode she shares her experience and important considerations for a successful transition to adult care in Duchenne muscular dystrophy (DMD). In the last decade, improvements in DMD care and therapeutic strategies have contributed to better patient outcomes and prolonged patient survival. Today,…
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Living with AADC Deficiency – Meet the Heger Family
Kelly Heger went back to school and trained as a nurse after her daughter Jillian was diagnosed with AADC deficiency (AADCd), a rare fatal pediatric disorder, over 26 years ago. Kelly was determined to understand the complicated medical language used by her daughter’s doctors and to play a pivotal role in the round-the-clock medical care…
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Guðjón CAN contribute to science & research in genetic disorders
Guðjón is from Iceland and is living with Duchenne. As an academically focused young man, he was always top of the class whilst studying for a degree in pharmacology at the University of Iceland. However, he realized that he could use his scientific talent and everyday experience of living with Duchenne to help support advances…
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Andrea CAN Find a Reason to Smile
Andrea from Sicily, Italy is an inspiring young man living with Duchenne muscular dystrophy. He knows that being optimistic and believing in oneself are fundamental to overcoming challenges and achieving great things in life. His story is proof that people with #DuchenneCan dare to be bold and make the most out of life. Andrea is…
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Our Commitment to the Rare Disease Community
PTC was founded in 1998 by a scientist with a desire to serve patients with rare diseases and unmet medical needs. Over the course of our history, we have grown to a team of more than a thousand employees and developed new innovative therapies. However, we’ve always maintained our foundational commitment to our patients and…
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Evolving Care in Duchenne Muscular Dystrophy (DMD)
In this podcast episode, our guest speaker Dr. Diana Castro, associate professor of pediatrics, neurology and neurotherapeutics at the University of Texas Southwestern, shares her extensive clinical knowledge and discusses the clinical, diagnostic and medical landscape in Duchenne muscular dystrophy (DMD). In addition, she speaks about management approaches in the care of DMD. Return to…
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Enrolling Your Child in a Clinical Trial – Get the Basics
Key Takeaways • PTC clinical trials are regulated and reviewed by the FDA in the United States, and by local regulatory authorities, ethics committees, and institutional review boards in respective markets • Most patients in PTC trials see an independent doctor for evaluation before enrolling • PTC trial staff and researchers monitor individual patient data…