The latest news from PTC

September 7 is World Duchenne Awareness Day, a global day aimed at raising awareness for those affected by Duchenne and Becker Muscular Dystrophy. This year’s theme is “Women & Duchenne,” and to honor this day, we’re highlighting inspiring women in the Duchenne community. Whether they are a patient, caregiver, or advocate, we celebrate their positivity,…

Those with Duchenne muscular dystrophy may face difficulties such as a limited social life, dependency on home care, and trouble accessing the same opportunities as able-bodied peers. These challenges have been found to have a negative impact on the psychological wellbeing of individuals with Duchenne. Accepting and coping with this kind of neuromuscular disability and…

MDA Hellas is a non-profit organization for people with Neuromuscular Disorders (NMD) in Greece, aiming to improve the quality of life of more than 1,500 affected families. Currently the organization aids in facilitating medical care for those with NMDs, offers psychological and social support, provides equipment and infrastructure to ease everyday routines, and raises public…

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…