Shalom’s rise to DuchenneCan
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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…
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Introducing 25 Remarkable Stories: Celebrating 25 People Who Impacted PTC
Twenty-five years. A quarter of a century. For PTC Therapeutics, our 25-year existence has been 25 years of bold, transformative science; of bringing moments to rare disease patients and their families. Twenty-five years of ups and downs, but 25 years of relentless optimism. Twenty-five years of people making PTC the company it is today. To…
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PTC Planting Trees for the Future
Last week in Switzerland, PTC colleagues teamed up to plant trees in an effort to make an environmental impact. In partnership with OneTree Planted and Almighty Tree this year, 100 Oak trees were planted to help combat climate change and prevent biodiversity loss for generations to come. Tree planting is critical to reforestation and afforestation…
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STRIVE: Living Full Lives – Achieving Your Aims in Transition to Adulthood
Pathfinders Neuromuscular Alliance is a UK-based, user-led charity that provides information, advice, and peer support to teenagers and adults with muscle-weakening conditions such as Duchenne. Pathfinders has uniquely positioned themselves as a non-profit both for and run by people with muscle-weakening disorders. All trustees and staff have muscle-weakening conditions and are committed to sharing their…
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Gabriela CAN inspire children to embrace their differences
Meet Gabriela, young and talented author of a children’s book called Timothy’s Magic Wheelchair. The book is all about a young man by the name of Timothy who never really liked his wheelchair and what it stood for. So much so, that he wasn’t sad or upset when it broke down. However, this all changed…
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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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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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Understanding Gene Therapy
At PTC, we study human genes with a mission to treat rare genetic disorders. Our scientists are excited about the possibilities of gene therapy when it comes to treating rare diseases. However, we understand that when people first think about gene therapy, they may have questions. What is it? How does it work? What problems…
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Employee Engagement: Alive and Well at PTC
For several years, PTC has worked with Gallup® – a global analytics and advice firm that helps leaders and organizations solve our most pressing challenges – to enhance employee engagement. Our global team is spread across more than 20 countries around the world, with some offices having just a small crew of mission-driven team members.…
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Targeting the Regulation of Protein Production for New Medicines
The complexity of protein production associated with protein disorders, especially from faulty genes, is formidable. But that hasn’t stopped PTC from innovating in the space of RNA targeting for novel medicines. In 2003, PTC discovered the first novel small molecule capable of suppressing premature termination of translation by allowing the ribosome to selectively read through…