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Inspiring “People of Determination” – #DuchenneCan

July 19, 2021

  1. Inspiring “People of Determination” – #DuchenneCan

    In the United Arab Emirates (UAE), people with special needs and disabilities are considered ”people of determination.” This designation is used in recognition of their achievements in different fields. Abdulla – who is 37 years old and living with Duchenne in the UAE – is one such incredible person who demonstrates determination in all parts…

  2. AADC Deficiency Awareness Day 2021

    Watch the October 22, 2021 Facebook LIVE Broadcast of AADC Deficiency Awareness Day In 2020, the AADC Family Network, in collaboration with PTC Therapeutics, MassBio and Boston Children’s Hospital, observed the first AADC Deficiency Awareness Day. AADC stands for aromatic L-amino decarboxylase – and AADC deficiency is a rare disease that affects the brain and…

  3. Play the Virtual Escape Game: Mission Duchenne™

    PTC presents an interactive, family-friendly event for escape game enthusiasts of all ages. Mission Duchenne™: A Virtual Escape game is intended to be a fun and empowering virtual game experience for families. The game content includes information about Duchenne muscular dystrophy. The game can be played as many times as you like. We invite parents…

  4. Pedro CAN Live Life to the Fullest – #DuchenneCan

    Pedro is from Campo Grande, Brazil and is living with Duchenne muscular dystrophy (DMD). He has many passions and interests that keep him energetic and happy. His love of writing has led him to a career in journalism, and he has even published two poetry books. Singing and composing are core passions and he has…

  5. Hope Is a Lifeline – A PKU Story

    Heidi Maxfield is mother to a son with phenylketonuria (PKU) and sits on the Board of Directors at Intermountain PKU & Allied Disorders (IPAD). The following article was contributed to the PTC Newsroom to help spread awareness about PKU. Reach Heidi on Facebook or Instagram @maxfam. Do you have a rare disease story to share?…

  6. Ashley Clarke: Remember the Caregivers

    Ashley Clarke is an HD caregiver from Northern Ireland. She has served as board member for the Huntington’s Disease Association of Northern Ireland (HDANI) and operates the #Imnotdrunk lifestyle blog for members of the HD community. Follow her on Instagram at @imnotdrunklifestyleblog, and Facebook @imnotdrunkblog. Do you have a rare disease story to share? Contact…

  7. Patrick CAN Realize His Dreams – #DuchenneCan

    Patrick – a.k.a. “Tick” – from São Paulo, Brazil, loves music and enjoys performing on stage. He is also living with Duchenne muscular dystrophy – but that does not define him. Instead, Tick is defined by his personality and his dreams. Watch the video to learn about Tick and how he is making his dreams…

  8. B.J. Viau: Turning Hope into Help for the Huntington’s Disease Community

    B.J. Viau is a lifelong Huntington’s disease (HD) advocate and has served as Board Chairman of the Huntington’s Disease Youth Organization (HDYO). The following article was contributed to the PTC Newsroom to help spread awareness about HD. Follow him on Twitter @BJsView. Do you have a rare disease story to share? Contact our news team…

  9. Amazon Duchenne Community CAN Count on Leandro – #DuchenneCan

    At PTC, we’re passionate about supporting those living with rare diseases, even when they live in the most inaccessible parts of the world. Leandro, a PTC colleague in Brazil, plays a key role in educating physicians in remote areas along the Amazon River. He shares information about Duchenne muscular dystrophy (DMD), and has helped make…

  10. Júlio CAN Engineer a Successful Future – #DuchenneCan