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DuchenneCAN on a NASA mission with Ethan

May 18, 2023

  1. DuchenneCAN on a NASA mission with Ethan

    Ethan Higginbotham’s passion for astronomy and physics is “out of this world.” His interest in all things outer space influenced him to pursue an internship with NASA, and one day, he hopes to broaden his astrophysics knowledge through completing a physics PhD. Ethan, who describes himself as determined and driven, shows that people living with…

  2. 25 Remarkable Stories: Tenacious Ferocity

    Jonathan Piacentino would tell you his proudest life moment was finally asking out a girl after working up the courage for many months. His mom, Christine, would disagree: “What about graduating college, isn’t that your proudest moment?” she asks him, laughing. Christine is personally very proud of her work with the Parent Project Muscular Dystrophy…

  3. Why We Must Consider the ‘Totality of Evidence’ for Novel Rare Disease Therapies

    Christian Werner, Executive Director, Global Medical Affairs, Global DMD Lead, PTC, believes the “totality of evidence” provides an important understanding of the impact of a unique treatment. In this article, he shares strategies for broadening the adoption of the totality of evidence. As a physician working in rare disease drug development, I am convinced that…

  4. Committed to Doing our Part: 2022 Corporate Sustainability Report Highlights

    Last week, we were thrilled to publish the PTC 2022 Corporate Sustainability Report. “Committed to doing our part” is a comprehensive look at our science, support for rare disease patient communities and access to care, our strengths-based learning culture, global equality, diversity and inclusion, efforts to help the environment, and much more. As a growing…

  5. Take on Duchenne Podcast: A Mother and Carrier of Duchenne Muscular Dystrophy Shares Her Journey

    Elizabeth’s journey with Duchenne Muscular Dystrophy (DMD) began when her son was diagnosed at the age of five. In this episode, Elizabeth reflects on the diagnosis of her son, her daughter’s and her own carrier diagnosis, family life, her coping mechanisms and management of the disease. Although her and her family’s lives have been impacted…

  6. Shalom’s rise to DuchenneCan

    Shalom Lim is living with Duchenne in Singapore. The sad loss of his brother to the condition and his experiences of neurodivergence as a disabled person has brought him adversity. However, now aged 27, he has learnt to view Duchenne as a companion and source of inner strength from his struggles. Following the loss of…

  7. 25 Remarkable Stories: Where Experience Can Make a Difference

    Luke Millington applied for an internship with PTC Therapeutics because he wanted to experience what it was like to work for a multinational company. He heard about PTC when his doctor mentioned their internship program was looking for applicants. After learning that PTC was a biopharmaceutical company focused on rare diseases, it seemed like it…

  8. DuchenneCAN & Sports proves to be a gold winning combination

    Powerchair Hockey is a team sport for people with physical disabilities who require a wheelchair to practice sport. The game is fast paced and intense…with two teams of five players battling against each other to score the most goals and win! The game prides itself on being very inclusive, with a team comprised of varying…

  9. Take on Duchenne Podcast: Cardiomyopathy Associated with Duchenne Muscular Dystrophy

    In this episode with Dr. Linda Cripe, we learn more about cardiomyopathy associated with Duchenne Muscular Dystrophy (DMD). We discuss care consideration and management strategies especially pertinent for manifesting DMD carriers and why screening and monitoring is so important. What more could be done to support carrier moms? Listen to the podcast to learn more.…

  10. Claudia Espinal – A Mompreneur with a DuchenneCan perspective

    Claudia is a mother of two, caregiver, social media content creator and Duchenne advocate from the Dominican Republic, living in New York. Originally a lawyer by trade, Claudia spent a happy, but demanding, few years raising her family whilst simultaneously juggling work commitments. However, this all came to a sudden halt when Claudia noticed that…