The latest news from PTC

Rare Disease Day, which takes place every year on the last day of February, is a global movement aimed at raising awareness of the 7,000+ rare diseases and more than 300 million people worldwide who live with a rare disease. With our deep experience in the rare disease space for more than 25 years and…

In this podcast episode we are discussing the topic of Nutrition and Duchenne with our guest speaker, Dr. Kindann Fawcett, Behavioral Health Scientist, Dietitian and Caregiver to her son with Duchenne Muscular Dystrophy.  Our conversation with Dr. Kindann Fawcett explores; Tune in to learn more about this topic. Visit the Take on Duchenne: North America…

Now 23, Luke Hains was 8 years old when his younger brother, Levi, was diagnosed with Duchenne muscular dystrophy. Looking after his brother and helping with his daily activities was part of life, even during college. While in the midst of figuring out the trajectory of his career during college, Luke realized that he could…

It is fair to say that Jessica Fabus Cheng is a very impressive woman who carries many crowns and titles including surgical nurse, sportswoman, mother, Duchenne carrier and cancer survivor, and Mrs. New York International 2023. She is also a former figure skater and in 2021 was listed as one of Taekwondo Life Magazine’s ‘10…

The PTC team in Brazil recently won three awards for their creative and impactful projects highlighting the rare disease community. First Up: The documentary series, “Living is Rare” (“Viver é Raro”) won the SABRE América Latina 2023 Award. The series was recognized with the award in the Public Education category. The world’s leading award in Public Relations, SABRE celebrates creative…

Felix Wu was four when he was diagnosed with Duchenne muscular dystrophy. Throughout his school career, he saw how Duchenne affected him, and how inclusive (or not) people could be. “People with disabilities have to think about so much more when we plan anything, just in terms of access,” Felix said. “At school, for example,…

The Patient Engagement (PE) team at PTC is passionate about education and support of a family’s rare disease journey, and this commitment includes support for patients and their families in the Spanish-speaking community. The team began meeting with the Spanish-speaking community in Puerto Rico more than five years ago, and realized there were minimal resources…

At 39 years old, there’s not much that Juan Morales hasn’t already achieved. Despite living with spinal muscular atrophy (SMA), a severe, genetic motoneuron disease causing muscle weakness and wasting, Juan’s own determination has led him to transform his life to find purpose. From writing an autobiographical book on personal development, advocating for people with muscular…

Judit Illes, Director, Government Affairs & Public Policy at PTC recently contributed to a newly published white paper, “Pioneering the New Era of Newborn Screening.” We connected with Judit to learn more about newborn screening, why it is so important and what she hopes people take away from this comprehensive paper on proposed reforms to…

In any conversation about patient advocacy for Duchenne muscular dystrophy, Pat Furlong’s name is bound to come up. Pat is the driving force behind Parent Project Muscular Dystrophy (PPMD), a patient advocacy organization founded by Pat and a group of parents of children with Duchenne in 1994. Since its founding, PPMD has played a pivotal…