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An innovative supply chain model for an innovative product

November 15, 2022

  1. An innovative supply chain model for an innovative product

    In July 2022, PTC’s first gene therapy and disease-modifying treatment for Aromatic l‐amino acid decarboxylase (AADC) deficiency was approved by the European Commission (EC). In this relatively new field of molecular medicine, PTC is one of the pioneers in the gene therapy, being the first therapy to be injected directly into the brain. Unlike the…

  2. Preparing Europe for a new generation of innovative therapies

    Thomas Bols, Head of Government Affairs and Patient Advocacy, EMEA and APAC at PTC, recently shared his perspective with Open Access Government on how preparing Europe for a new generation of innovative therapies has the potential to transform the lives of patients. Gene therapies are bringing hope to patients living with ultra-rare diseases. In the…

  3. Adam Peska: Boccia Paralympic gold medalist from Czech Republic

    Adam Peska is an athlete from Czechia who won gold in Boccia at the 2020 Summer Paralympics in Tokyo. He is the second person ever with Duchenne Muscular Dystrophy to win a gold medal in any event at the Paralympics. He has also won the Best Male Debut athlete at the 2021 Paralympic Sport Awards,…

  4. Honoring AADC Deficiency Awareness Day 2022

    For the third year, PTC will commemorate AADC Deficiency Awareness Day on Sunday, Oct. 23. This groundbreaking event was established in 2020 and spearheaded by AADC deficiency caregiver and founder of the AADC Family Network, Kelly Heger. The official designation of AADC Deficiency Awareness Day has enabled parents and caregivers, patient advocacy groups, legislators and…

  5. Lukas Fischer supporting people with Duchenne to live self-determined lives

    The Mathilde Escher Foundation was founded in 1865 and provides specialized housing, training, work, and leisure opportunities for people with muscle-wasting diseases in Zurich, Switzerland. Lukas Fischer is the Head of Communications and Head of the Graphic design workshop at the foundation. He is committed to the foundation’s goal of supporting people with Duchenne, and…

  6. PTC Measures Moments on World Duchenne Awareness Day

    Each year on September 7, the Duchenne muscular dystrophy (DMD) community unites to raise awareness for the 250,000 people around the world living with DMD. This year was no different, and PTC was proud to join the many supporters and advocates globally on September 7 to show support for the DMD community. This year’s theme…

  7. Benjamin James – Living with Duchenne means you CAN live the life you want

    Benjamin James has a first-class honors Neuroscience degree; he is also a patient advocate and a healthcare communications consultant. When he was four years old, Benjamin was diagnosed with Duchenne, which was managed in his younger years with steroid treatment, exercise and a good physiotherapy routine. As he reached his teenage years, Benjamin began attending…

  8. World Duchenne Awareness Day: Celebrating Women in Duchenne

    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,…

  9. 2022 STRIVE Award Recipient: Neuromuscular Disorders Association of Turkey/KASDER

    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…

  10. 2022 STRIVE Award Recipient: MDA Hellas – Muscular Dystrophy Association of Greece

    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…