terri ellsworth
Contact Terri
Terri Ellsworth is a seasoned and proactive advocate/activist from Pittsburgh, Pennsylvania and is devoted to raising awareness for Duchenne muscular dystrophy and all rare disease.

She has one child, her son Billy, 19 years old, with Duchenne Muscular Dystrophy, the most lethal genetic childhood disease. Terri and Billy were frontline advocates for approval for the first drug ever to receive FDA approval for muscular dystrophy. Billy was one of 12 boys in the United States and the only boy in Pennsylvania to trial his treatment for 5 1/2 years before receiving FDA approval in September 2016. Billy is still fully ambulatory, independent, and self-sufficient thanks to his weekly infusion treatment of Eteplirsen, now Exondys51. They both testified to the FDA on behalf of Billy's drug treatment. He has been a trailblazer and pioneer for medical science and innovation and continues to defy his disease.

Terri was a committee member, Biomarkers 2, for the first ever FDA Duchenne Draft Guidance in 2014. She also served as a Community Advisory Board Member for this same guidance. She has reviewed extensive IRB consents and has participated as a community member for observation at local IRB committee meetings.

She is an RDLA Rare Disease Legislative Advocate and advocates on Capitol Hill for legislation for rare disease patients. Terri also serves as mentor and support for newly diagnosed families both domestically and abroad. She has a respected and credible name and voice in the rare disease community and is often requested to speak to various audiences which have included University of Pittsburgh's Medical School, graduate students in the Genetic Counselors Program,  and MDA events to name a few. Terri and Billy were also requested to speak and present an Innovation Award in 2017 at NORD's gala event in Washington, DC to the CEO of Sarepta Therapeutic. They were also invited to Perth, Western Australia in 2017 and 2019, where Billy's drug treatment was developed. While there, they met and spoke with dignitaries, scientists, and academia.

Terri also serves as a Citizen Scientist for OneFlorida Clinical Research Consortium at  University of Florida and is also a RUGD Ambassador (Rare and Undiagnosed Genetic Disease) for Illumina Biotech. She is also a PALS Ambassador (Patient Airlift Services).

In addition to Terri's full time advocacy work and volunteering endeavors, she is a very seasoned Interior Designer specializing in kitchens and baths and has studied at University of Pittsburgh.
Most recently, Terri collaborated with the pulmonology department at Children’s Hospital of Pittsburgh to form the Neuromuscular and Family Medical Advocacy and Advisory Council, the first of its kind which is committed to partnering community, family and medical advocates for the benefit of neuromuscular patients at Children’s Hospital of Pittsburgh. Terri’s role is a permanent non-rotating liaison and co-chair.

Areas of Advocacy Expertise:

  • Pharma/Biotech Advocacy

  • Whitepaper Development

  • Social Media & Marketing

  • Story Telling

  • Advocacy Partnerships outside of disease-specific community

  • Community Engagement

  • Public Speaking

  • Legislation/Lobbying in Washington D.C., Capitol Hill and locally in Pittsburgh

  • LinkedIn - White Circle

The Rare Advocacy Movement (RAM) is a patient advocacy initiative focused on documenting the evolving complex structure and unique dynamics of the rare disease patient advocacy landscape. RAM serves as a Center of Insight (COI) for those interested in participating in collaborative patient advocacy initiatives and working groups within the rare disease industry. The RAM membership is comprised of a network of patient advocacy professionals that have vowed to abide by the established Code of Conduct for Membership.