Stay Small in Mission, Even as You Grow Big

When you start a rare disease foundation, everything is personal. It's a living room operation, run by parents with ringed eyes and raw hearts, huddled around laptops trying to translate grief into change. You’re small, but your mission is massive. You want to move mountains—for your child, for all our children.

But here’s the truth no one tells you: the more successful you become, the more danger there is of drifting. Away from your “why.” Away from your purpose. And worst of all—away from the patient.

Start with Fire. Stay with Purpose.

In the early days, no one has time to posture. You're just trying to survive the hospital visits, the insurance denials, the specialists who can’t pronounce your child’s disease. The mission is clear: keep your kid alive. Keep others from suffering.

But when growth comes—recognition, funding, board members with big resumes—it’s easy to slip into respectability politics. Polished PR replaces raw truth. Meetings multiply. Metrics matter more than moments.

Suddenly, you're speaking about patients instead of for them. Or worse—instead of them.

That’s the beginning of the end.

Guiding Principles for Staying Grounded

Here are the truths I’ve learned the hard way—from leading small, and navigating the risks of becoming “big.”

1. The Patient Voice Is Not Optional. It's the North Star.

In ultra-rare disease, I’ve sat through hundreds of meetings where my presence checked a box. “Caregiver input—check.” No one asked for my insight. No one followed up. I was there to be seen, not heard. I’ve stood in photo ops with Senators, posed next to FDA officials—moments used to say, “Look, we met with ultra-rare disease.” But after the flash, nothing changed. The headlines didn’t match the policy. The promises didn’t deliver outcomes. The urgency vanished with the camera crew. We don’t need visibility without action. We don’t need seats at the table if our voices aren’t respected.

That’s not inclusion. That’s tokenism.

Every decision, partnership, and press release should be able to stand in front of the community and say, “We did this for you.” And mean it.

If you're building anything for rare disease—legislation, research, treatment—you should be asking:

• Would a patient feel proud of this?

• Would a caregiver feel seen?

• Would someone living this life believe we had their back?

If the answer is no, it’s time to go back and do better.

Patients and families aren’t lucky to be in the room. They are lucky to have them. Their lived experience isn’t a nice-to-have. It’s the most valuable expertise.

So treat it that way. Center it. Respect it. Compensate it.

Because if you’re building without them, you’re building it wrong.

2. Money Should Fuel the Mission—Not Become It.

Yes, you need funding. But if your foundation starts chasing donor dollars more than justice, it’s time to stop and recalibrate. Your bank account should never define your values—your values should guide every dollar you spend. I’ve done more with grit, truth, and persistence than many have done with millions. I’ve had no budget—literally nothing—and yet I’m still here, still in the fight, and respected because of it. Money helps. But never forget: your voice, your story, and your integrity are worth more than any check. That’s what truly moves the needle.

3. Professionalism Is Good—Until It Muffles the Truth.

It’s good to be polished—but not if it means you stop telling the truth. As your advocacy grows, don’t lose the realness and urgency that got you here. Yes, experts and consultants can help. But if they start watering down your message or making you sound too safe, that’s a problem. In ultra-rare disease, there aren’t many true experts. Most of the time, the people who know the most—and care the most—are the ones living it: parents, patients, caregivers. Your story matters. Your truth matters. And in rare disease, it’s one of the strongest tools you have. Sometimes, outside consultants (especially ones tied to pharma) will try to clean up your message too much. They’ll tell you to be “neutral” or play it safe. That might get you into some meetings, but it won’t bring real change. Instead, look for people who are honest, smart, and not connected to industry money. You need support from those who can help you stay focused and bold—not ones who turn down your volume. In rare disease, we don’t have time or data to waste. The truth can be uncomfortable—but it’s also what moves things forward. If your voice rattles the system a little, good. That’s how change starts.

4. Sponsors Are Partners, Not Puppet Masters.

Pharmaceutical companies often fund rare disease advocacy. That support can be life-changing—but it can also be compromising if you're not careful. Just because a company offers sponsorship doesn’t mean they get to write your script. Your community’s trust is not for sale. Set clear boundaries. Be grateful for support—but fiercely protective of your independence and the patient voice. The National Orginization for Rare Disease (NORD) is developing a standardized template to guide interactions between patient advocacy groups and the pharmaceutical industry. This initiative aims to address the complexities and potential conflicts of interest inherent in such collaborations, ensuring that patient voices remain central and uncompromised. The template is expected to be released within the next year. Stakeholders are encouraged to stay informed and participate in its development to promote transparent and ethical partnerships that prioritize patient needs.

5. Coalitions Are Powerful—but Don’t Lose Yourself.

Joining national coalitions and umbrella advocacy groups can help amplify your voice—but only if you stay true to your own. Ultra-rare diseases often face different hurdles, and what works for one condition might not fit yours. Align where it makes sense, but don’t mold yourself to someone else’s legislation if it doesn’t serve your community. Also, be wary of any organization that tries to control your narrative. I've heard of advocates being told they can’t speak to their own senators or congressional reps without group permission. That is false. You are a constituent—you can always reach out to your representatives. My rule? Advocate for what you need. If you’re supporting a specific legislative ask, yes—certain parts of your story may matter more. But no group should ever silence you or own your voice.

6. Never Forget the Families Still in the Fire.

There are parents right now who are where you were—fighting for a diagnosis, begging for a therapy, rationing medical food because insurance won’t pay. Let them be your compass. If your foundation isn’t making their burden lighter, then who is it helping? Ask what is the goal and out come of each action. Will it help patients?

7. Start Building Your Power Before the Policy Exists.

You don’t need a bill to start collecting patient stories. You don’t need a legislative agenda to begin a constituency list. Do it now. Trust me—you’ll need it later. I started with ten moms. That grew into hundreds. Then thousands. It’s not just a list—it’s your leverage. Start a excell spread sheet or a google form to keep track. I promise you you will need this. Even if its 7 patients it matters.

What’s At Stake

For ultra rare, the cost of losing focus isn’t just philosophical—it’s life and death. A single newborn screening delay can cause irreversible brain damage. One denied therapy means another child loses speech or mobility. The lifetime burden per patient for ultra rare —avoidable, if we had earlier diagnosis, approved treatments, and functional policy support. But systems only change when foundations stay brave. Stay truthful. Stay rooted in the real stories—not just the ones that fit neatly into grant applications.

To the Next Generation of Founders

If you're starting a rare disease foundation, I offer you this:

Stay wild enough to scare the status quo.
Stay grounded enough to walk with your community, not above it.
Stay principled enough to say “no” to what looks shiny but isn’t sacred.
And stay small in heart—even when you grow big in influence.

Because the bigger you get, the more the patient voice must rise. That’s how we win.