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…

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…

Some of the most memorable and formative times in Arya Singh’s childhood were spent participating in clinical trials. Her parents enrolled her in a clinical trial for a treatment for spinal muscular atrophy (SMA), a severe, progressive neuromuscular disease, when she was ten years old. She grappled with some tough experiences in the trial –…

Loren Eng didn’t set out to run a non-profit. That changed when her daughter, Arya, was diagnosed with spinal muscular atrophy (SMA), a severe, progressive neuromuscular disease, when she was about two years old. Arya was a beautiful baby and developing perfectly, but soon Loren recognized there was something wrong: Arya wasn’t walking properly, and…

People living with rare diseases have many strengths, abilities and ambitions. One of them is playing and engaging in sports. This October, the 2023 FIPFA Powerchair Football World Cup is taking place in Sydney, Australia and the world’s best powerchair footballers are coming together to compete for the 2023 trophy. PTC is proud to support…

For PTC, 2022 was a milestone-reaching, patient-supporting, award-winning, employee-engaging, community-driven year. It was also another year of many firsts. The first approved disease-modifying treatment for AADC deficiency, and the first marketed gene therapy directly infused into the brain, was approved in Europe and the UK. We also had the first global regulatory approval for treatment…

Powerchair hockey is a team sport played by athletes who require the use of an electric wheelchair in their day-to-day lives. Athletes have disabilities ranging from Muscular Dystrophy or SMA, to cerebral palsy or severe spinal cord injuries. Learn More about the 2022 IWAS Powerchair Hockey World Cup Meet the Swiss National Team

”Your child is severely physically disabled” – after giving this kind of diagnosis, physicians are faced with an important task: to give desperate parents some perspective. Impossible? Not at all! Because even with a severe disability, so much is possible – sometimes even a world championship title. Let yourself be inspired. Sport at the highest…

Powerchair hockey is a team sport played by athletes who require the use of an electric wheelchair in their day-to-day lives. Athletes have disabilities ranging from Muscular Dystrophy or SMA, to cerebral palsy or severe spinal cord injuries. Learn More about the 2022 IWAS Powerchair Hockey World Cup

At PTC Therapeutics, we continue to be inspired by the communities we serve. We are proud to be sponsoring this year’s IWAS Powerchair Hockey World Cup, which is taking place from the 9th – 14th August 2022, in Switzerland. The IWAS Powerchair Hockey World Cup is the most important international competition for powerchair hockey athletes.…