From Grief to Advocacy: Amy Fadden - Rare Disease Advocate | Co-Founder and Vice President Aislinn's Wish Foundation
In October 2022, the earth cracked open and inhaled everything solid, leaving me unmoored, terrified, and torn apart with grief. My daughter, just a few weeks past her twelfth birthday, died from a rare genetic disorder.
Sanfilippo Syndrome is a lysosomal storage disorder that causes progressive neurological damage, stealing children’s abilities like walking, talking, and eating. It’s always fatal, and there is no treatment or cure. We knew this, and we clung to every day with Aislinn, at the same time raising money to fund research that we fervently hoped would save her. Despite everything we still weren’t ready. We would never have been ready.
For 12 years, Aislinn and our son, 2 years older, were my purpose. Once Aislinn was diagnosed, at age 4, our lives became a mix of fighting to help her maintain her skills, allowing her to be the vibrant child she was, and doing all the normal things families do. As Sanfilippo took a greater toll, her needs increased. She lost her speech, couldn’t stand on her own, and had a feeding tube.
It was exhausting, for her and for us, and I would do it all over if it meant she were still here.
When your purpose is whisked away in a breath, it leaves you tumbling in a turbulent storm. Several months after Aislinn died, I sent a message to a friend who had been a patient advocate at one of the companies researching gene therapy for Sanfilippo. She replied with a link to a training programme through the Professional Patient Advocates in Life Sciences (PPALS).
The training was for patient advocates in non-profit organizations and biotech/pharmaceutical companies. At a time when nothing felt right, the training offered direction.
PPALS: A Closer Look
The PPALS Patient Advocate Program is a pillar of the broader non-profit PPALS organization, which is committed to promoting the role of Patient Advocacy within the biotech and pharmaceutical industries. The goal is clear and unwavering: to foster an environment where individuals are empowered and trained to significantly enhance the lives of their respective patient communities.
Through this program, advocates are equipped with the tools and knowledge required to bridge the often-divergent worlds of patient needs and drug development. This is achieved via a multifaceted approach:
In May, I traveled to Sioux Falls, South Dakota, for the Certificate in Patient Advocacy training. Over several days, we heard from leaders in advocacy about how to make patient voices heard in drug development.
Aislinn never qualified for clinical trials, and it was an ongoing frustration for us. Trial designs and endpoints didn’t seem to align with what we as parents considered a successful outcome.
This is true among many patient populations, especially in rare diseases, and the PPALS presenters showed us how to navigate a landscape of nonprofits, industry, and policy. Their knowledge was impressive, and the commitment PPALS faculty and attendees demonstrated to rare disease communities ignited a spark of excitement I hadn’t felt in months.
This was a purpose I could pursue, one I knew Aislinn would condone. All the years of advocating for Aislinn and the connections I made along the way found a meeting place, and I left Sioux Falls with a lighter heart.
Aislinn’s light will continue to shine, as I use it to illuminate the faces of patients who still fight for their futures.
Amy Fadden is Co-Founder and Vice President of Aislinn's Wish Foundation. With extensive knowledge of how chronic illness impacts children and their caregivers, Amy has the unique ability to understand the perspective of both patients and providers and to work with medical teams and community agencies to effectively and compassionately address the needs of patients.
We would like to thank Amy for sharing her very personal experience of the fight advocating for Aislinn and the value of the PPALS Patient Advocate Programme.