Skip to main content

Elijah CAN “Destroy Duchenne” – #DuchenneCan

October 6, 2021

  1. Elijah CAN “Destroy Duchenne” – #DuchenneCan

    Elijah, from California, refuses to let Duchenne muscular dystrophy define him and his future. When Elijah was only 15 years-old, he founded Destroy Duchenne, a non-profit organization that aims to ”complete the cure” for Duchenne by advancing gene-editing and gene therapy technologies into human practice. His driving force is to not only bring hope to…

  2. Duchenne United at PTC

    This year’s World Duchenne Awareness Day, our proud employees worldwide united in their efforts to mark this significant day with various disease awareness and educational activities. With the focus on ‘Adult Life & Duchenne’ we had a chance to engage and collaborate with a number of young men and adults living with Duchenne to better…

  3. Duchenne Diagnosis… Reflections of a Father, 28 Years Later

    This is the story of Filippo Buccella. But this is also the story of his son, Luca, who was diagnosed with Duchenne muscular dystrophy (DMD). A childhood diagnosis changes more than just a patient’s life. It changes the lives of everyone around them. Watch their story:

  4. Life with Leigh Syndrome – Meet Brent, Suzette and Chloe

    Brent and Suzette Fields learned just what the phrase “don’t sweat the small stuff” means when their lives changed due to a rare disease diagnosis. Early in her first year of life, their daughter Chloe showed signs of some developmental delays which led them to seek physical therapy for her. After a few sessions, the…

  5. For L.A.N.D., Community CAN Feel Like Family – #DuchenneCan

    “L.A.N.D. is more than an organization, it’s like a family. We support each other during challenging times and we celebrate together our victories.”  ~ Founder of the Lebanese Association for Neuromuscular Diseases, Aida Aida and her husband founded the Lebanese Association for Neuromuscular Diseases (L.A.N.D.) in 2011, following their son Wafik’s diagnosis with Duchenne muscular dystrophy.…

  6. Adult Life and Duchenne Podcast – #DuchenneCan

    In this episode, we meet three fantastic, young men who live with Duchenne muscular dystrophy, and who, by following their interests, have developed exciting lives and careers. Björn Jönsson is self-employed with a strong commitment to the Swedish Foundation for Muscular Dystrophy Research, among other causes. He also works with gaming development under the motto…

  7. Bringing Together the Global PKU Patient Community

    In 2021, PTC is a silver sponsor for the Global PKU Patient Conference in September. We had a chance to catch up with Monique Cooper – the conference chairwoman and president of the Metabolic Dietary Disorders Association in Victoria, Australia –  to learn a little more about phenylketonuria (PKU) and how this patient-facing conference plans…

  8. STRIVE: Structuring the Clinic for Basic Care at ASCADIM

    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 and trusted information. Via ASCADIM, patients and their families can access vital support, guidance, and free physical therapy –…

  9. A Letter to the AADC Deficiency Community

    Dear AADC deficiency community, For more than 20 years it has been our mission to bring life-changing medicines to patients with rare disorders. In these challenging times, we are even more committed to ensuring that patients have access to safe and effective treatments. PTC-AADC is an investigational, one-time gene replacement therapy that, if approved, will…

  10. People with Special Needs CAN Travel – #DuchenneCan

    Sebastien is the founder of Handiscover – an online hub for booking accessible travel accommodations. He’s passionate about travel and has lived all over the world. He was determined that he’d keep his travel habits once he had children. However, at four years-old, his son Teo was diagnosed with Duchenne muscular dystrophy, and Sebastien soon…