The latest news from PTC

When the Associacao Cearense de Distrofias Musculares (ACDM) was founded in 2017 they had a mission; to provide access to therapies and to share parental guidance on muscular dystrophies, including Duchenne, to those affected in the north and northeast regions of Brazil. These regions have a higher prevalence of genetic disorders, and of Duchenne in…

Action Duchenne is a charitable organization with a clear vision: a world where lives are no longer limited by Duchenne. They were founded with three core objectives: (1) develop effective treatments for all by funding research, supporting clinical trials, and campaigning for access; (2) build a community by uniting and supporting families, educating about Duchenne,…

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…

Stephen Keane, Associate Director, Talent Acquisition & Strategy, is about to embark on a six-day, 761-kilometer cycling event to raise money for Muscular Dystrophy Ireland. The challenging cycle will start in Paris and will head south to finish in Nice and will encompass some of the toughest hill climbs in Europe. As an active cyclist,…

Due to medical advances and evolving disease management, people with Duchenne are now living longer than before. This new reality creates new opportunities for independence and aspirations, previously unthinkable for those living with the disease and their families. Whether that education, sport, work or simply having more autonomy over their day to day lives. But…

ASCADIM provides physical, psychological, and social care to people with muscular dystrophies in Southern Brazil. The organization was created based on insights from parents on the despair they felt following diagnosis and their desire for more trusted information. Via ASCADIM, patients and their families can access vital support, guidance, and free physical therapy – crucial…

Treatments for rare diseases, such as gene therapies, are complex, requiring a high level of clinical expertise and often administration in a specialist center. As these diseases are not common, treatments and specialist centers are not accessible in every country in Europe. Patients with rare diseases may therefore have to cross geographical borders to receive…

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