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AADC Deficiency Awareness Day

October 6, 2020

  1. AADC Deficiency Awareness Day

    The debilitating rare genetic disorder aromatic l-amino acid decarboxylase (or AADC, for short) deficiency is the focus of the first AADC Deficiency Awareness Day in the State of Massachusetts, United States on October 23, 2020. This official designation will help enable parents and caregivers, patient advocacy groups, legislators, and gene therapy experts to raise awareness,…

  2. PTC’s Mary Frances Harmon Named to PharmaVOICE 100

    PTC SVP, Corporate and Patient Relations Mary Frances Harmon has been named to the 2020 PharmaVOICE 100, an annual list of the most inspiring people in the life sciences industry for her work in patient advocacy. PharmaVOICE caught up with Harmon in a video interview to discuss her legacy as a patient champion. She speaks…

  3. STRIVE Awards Sponsor Hope Online

    “For over 30 years, it has been ADM’s mission to improve the quality of life for the people with neuromuscular diseases in Argentina. After winning this year’s STRIVE awards, we are overjoyed to be able to develop an online community which will continue to support and address the needs of the 1,600 Duchenne patients in…

  4. Education for Duchenne Caregivers in Hungary

    “For many parents of Duchenne patients in Hungary, these events provide a trusted environment where they can share their concerns, and experience a few well-needed hours of respite as their child takes part in many fun activities with their peers.” – Dr Gabriella Oberrecht, Chairperson of the Board, Healing Goodwill Foundation – Duchenne Hungary Receiving…

  5. Counseling Duchenne Patients in Russia

    “With so many individuals providing care to their child alone, without professional help, the ‘Not Alone’ initiative, made possible by the STRIVE program, will provide an invaluable support system to families impacted by Duchenne muscular dystrophy.” – Margarita Urmancheeva, President, GAOORDI Due to a lack of funding, only a few specialist care facilities for Duchenne…

  6. PTC Grant Funds Life Science Immersion Program

    “The next young generation can play a pivotal role in supporting the rare disease community. With this year’s STRIVE Award, we can equip students with the essential tools and knowledge needed so they can be young Duchenne ambassadors in their community.” – Richard Horgan, Founder and President, Cure Rare Disease The rarity of diseases such…

  7. Launching a D&I Program at Our Rapidly Growing Company

    I am Martin Rexroad, senior vice president in Human Resources for PTC Therapeutics. I want to introduce and explain our new Diversity and Inclusion (D&I) program and how it will advance what we have always been doing at PTC – now for a much larger team. Q: Why is now the right time for a…

  8. PTC Fellowship Program Aims to Help Recent Grads

    We know your chances of obtaining a well-paying job go up with a college degree on your wall. However, Pew Research Center concludes that the 2020 employment rates due to the COVID-19 (coronavirus) pandemic mirror those of the Great Depression. The pandemic is making it extremely difficult for recent college graduates to enter the workforce…

  9. PTC Celebrates Rare Disease Day 2020

    This February, PTC celebrated Rare Disease Day on a global scale, with employee participation in disease and patient awareness, as well as displays of support for the rare disease community we serve. These images demonstrate our ongoing commitment to bringing more of life’s precious moments to patients with rare diseases and their loved ones. From…

  10. PTC Awards Grants for Global Duchenne Muscular Dystrophy Research

    In 2019, we launched PRIORITY, a global innovative research funding program to support pioneering clinical research initiatives that address the unmet medical needs of patients with the rare genetic disorder Duchenne muscular dystrophy. The goal is to advance the diagnosis and care of patients and support unique research programs that are focused on increasing the number…