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PTC in 2021: A Year of Expansion

December 22, 2021

  1. 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…

  2. 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,…

  3. 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…

  4. 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…

  5. 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…

  6. 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…

  7. Elijah CAN “Destroy Duchenne” – #DuchenneCan

    Elijah, from California, refuses to let Duchenne muscular dystrophy define him and his future. When Elijah was only 15 years-old, he founded Destroy Duchenne, a non-profit organization that aims to ”complete the cure” for Duchenne by advancing gene-editing and gene therapy technologies into human practice. His driving force is to not only bring hope to…

  8. Duchenne United at PTC

    This year’s World Duchenne Awareness Day, our proud employees worldwide united in their efforts to mark this significant day with various disease awareness and educational activities. With the focus on ‘Adult Life & Duchenne’ we had a chance to engage and collaborate with a number of young men and adults living with Duchenne to better…

  9. Gaming with the Guys with Duchenne – DUG 2021

    PTC is excited to partner with CureDuchenne again in 2021 for the Duchenne United Gaming (DUG) Tournament – happening during the CureDuchenne 2021 FUTURES on October 8 and 9! Over the years, patients with Duchenne muscular dystrophy have utilized online gaming as a means to socialize and have fun from the comfort of home. As…

  10. Duchenne Diagnosis… Reflections of a Father, 28 Years Later

    This is the story of Filippo Buccella. But this is also the story of his son, Luca, who was diagnosed with Duchenne muscular dystrophy (DMD). A childhood diagnosis changes more than just a patient’s life. It changes the lives of everyone around them. Watch their story: