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News About PTC Gene Therapy Published by the European Molecular Biology Organization (EMBO)

October 25, 2021

  1. News About PTC Gene Therapy Published by the European Molecular Biology Organization (EMBO)

    PTC is excited to announce the publication of “Gene therapy in the putamen for curing AADC deficiency and Parkinson’s disease” in the EMBO Molecular Medicine journal online with open access. Read the Article Featuring collaboration from medical experts Paul Wuh-Liang Hwu, Karl Kiening, lrina Anselm, David R. Compton, Takeshi Nakajima, Thomas Opladen, Phillip L. Pearl,…

  2. Expanding the Rare Disease Patient Voice at the BIO Patient & Health Advocacy Summit

    For the past three years, PTC has joined patient advocacy organizations, academia, regulators, the life sciences industry, and other stakeholders from around the United States as a sponsor of the BIO Patient & Health Advocacy Summit. This two-day inspirational event offers PTC employees the chance to join their solution-focused peers and share challenges, ideas, and…

  3. Enrolling Your Child in a Clinical Trial – Get the Basics

    Key Takeaways • PTC clinical trials are regulated and reviewed by the FDA in the United States, and by local regulatory authorities, ethics committees, and institutional review boards in respective markets • Most patients in PTC trials see an independent doctor for evaluation before enrolling • PTC trial staff and researchers monitor individual patient data…

  4. 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…

  5. Gaming with the Guys with Duchenne – DUG 2021

    PTC is excited to partner with CureDuchenne again in 2021 for the Duchenne United Gaming (DUG) Tournament – happening during the CureDuchenne 2021 FUTURES on October 8 and 9! Over the years, patients with Duchenne muscular dystrophy have utilized online gaming as a means to socialize and have fun from the comfort of home. As…

  6. 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…

  7. 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.…

  8. 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…

  9. 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 –…

  10. 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…