My interactions with Pat Furlong, mother of two Duchenne boys and Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), are generally clinical. What are the newest trends in science? What are the promising pharmaceutical development programs? What’s going on at the FDA?
These conversations are robust but Pat and PPMD are about more than that. Every time we speak, Pat reminds me her organization begins with the word “Parent.” Even though PPMD is a parent advocacy organization with a highly sophisticated understanding of what it takes to bring new therapies to market, it’s heart and soul (and Pat’s heart and soul) isn’t clinical – it’s practical. PPMD isn’t just about advancing science and drug development, it’s about personal development. The journey of parents and families. Pat Furlong personifies the phrase “patient-focused drug development.”
Drug development is a journey. Long, arduous, expensive, frustrating. Being a parent of an individual diagnosed with Duchenne is all of those things – with an even more important measure – love and devotion. If you think that sounds soft and sappy, please disabuse yourself of that notion. It’s the most crucial dimension. And there is no endpoint.
But is this relevant for developers? For their stockholders? For financial analysts? For the FDA? The answer is a resounding “yes” – but many developers don’t know it, don’t recognize the value of the patient voice in both clinical development and the FDA regulatory process. Without regular and robust alliances with the patient community (beyond traditional and very transactional post-approval marketing partnerships), there are very few regular and robust relationships. These ghettoized relations leave potent opportunities untapped – until it’s too late. “Too late,” because what may seem to be a trivial benefit to a risk-averse FDA reviewer (such as the ability for a Duchenne’s child to scratch an itch in the middle of the night) can be highly relevant to the parents of that boy’s parents. The moral of the story is that the time for a developer to build alliances isn’t when they receive a Complete Response letter or when the marketing plan is due.
Such consideration isn’t unique to the Duchenne’s community. PPMD (together with forward-thinking regulators like Dr. Janet Woodcock) invented the concept of patient-driven FDA guidance development. And it’s important to remember that the driving imperative wasn’t just to develop a more modern and streamlined regulatory process – but new ways to think about risk/benefit.
Pat Furlong puts it all in the proper perspective – the patient perspective. Here it is in her own words.
A diagnosis of Duchenne muscular dystrophy is traumatic—full stop. It does not matter whether the child is a newborn, two or ten, whether the diagnosis is expected or comes out of nowhere. In that moment, something fundamental breaks open. Parents, siblings, grandparents, and all who love that child are thrust into a reality they did not choose and could never have imagined.
We are often told that grief follows a process. That after shock and sadness comes acceptance, as though there is a finish line where the pain resolves and life settles into a new normal. I fundamentally disagree with this framing. Duchenne is not a single loss to be mourned and then set aside. The diagnosis represents the loss of what was hoped for, planned for, assumed. Futures imagined with ease—independence, longevity, simplicity—are suddenly no longer available in the way they once were.
This is not a grief that ends. It is grief that becomes a state of being.
From the day of diagnosis forward, grief does not disappear; it is remodeled. Again and again. As life evolves, as the child grows, as new challenges and new possibilities emerge, grief is reshaped to fit a constantly changing world.
For me, the experience is best compared to buying a hundred-year-old home. At first glance, it may look charming, even livable. But very quickly, you realize that everything needs work. You begin with one room—focusing your energy on what is most urgent. You repair, update, reinforce. Then you move to the next room, and the next. Years pass. The work is ongoing. And the truth is that it’s never finished.
Grief in Duchenne works the same way. Early on, it may center on the diagnosis itself. Later, it may be about mobility, about cardiac or respiratory function, about the growing awareness of limitations that peers do not face. At each stage, families adjust expectations, recalibrate hopes, and renegotiate dreams. The remodeling continues.
Importantly, progress does not erase grief. Advances in care, opportunities to participate in clinical trials, and access to therapies can bring real hope—and they should be celebrated. But each new opportunity also requires another round of emotional reconstruction. What does this treatment mean – and not mean? What doors might open and which remain closed? With every gain comes another set of “what ifs.” Another reworking of the internal landscape.
The underlying burden remains. It is carried quietly, often invisibly, by families who are told—implicitly or explicitly—that they should be further along in their acceptance by now. This expectation misunderstands the nature of living with a progressive, life-limiting disease. There is no static endpoint where grief is resolved because the disease itself does not stand still.
And yet, this ongoing grief does not preclude joy, purpose, or fierce love. Remodeling a home does not mean you stop living in it. You find beauty where you can. You celebrate what works. You take pride in what you’ve built, even as you acknowledge what still needs repair. Families living with Duchenne do the same. They love deeply, advocate relentlessly, and adapt continuously—all while carrying a grief that never fully lifts.
We do families a disservice when we frame acceptance as an outcome rather than an ongoing process of adjustment. Grief is not something to “get over” in Duchenne. It is something to live with, to reshape, and to carry forward as the world changes.
Recognizing this truth does not make families weaker. It makes them honest. And it creates space for compassion—not just at diagnosis, but at every stage that follows.
Pat reminds us (and we need regular reminding) that all things considered, more timely and dimensional relationships between developers and the patient communities they serve (note: not the other way around) are important and must be nourished. It’s good science, savvy regulatory strategy, and smart marketing -- in that order. It’s the right thing to do and it’s good for business. A terrific healthcare double-play. Developers who fail to recognize and actuate this hierarchy of patient involvement put their own development programs at risk.
Pat Furlong is Founding President and CEO of Parent Project Muscular Dystrophy
Peter J. Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest and a visiting professor at the University of Paris School of Medicine.
