Introducing #ExtraordinRARE 2025: A Bold New Rare Disease Week Expedition

Every journey begins with a single step. For the rare disease community, that step is often taken against formidable odds—navigating a landscape where too many remain overlooked, underfunded, and left waiting for breakthroughs that don’t come fast enough. Here at Emily’s Entourage (EE), we’re not just walking this path. We’re blazing a new trail.

This Rare Disease Week, we launch the 2025 #ExtraordinRARE campaign; a reflection of EE’s unyielding commitment to leading the charge to redefine what’s possible for rare diseases, most especially the final 10% of people with cystic fibrosis (CF)—a rare group within a rare disease! This year, our mission is clear: we are not waiting. We are pushing boundaries, driving scientific innovation, and leaving no stone unturned to ensure that no one with CF is left behind.

Rare Doesn’t Mean Small

A rare disease is any disease that affects fewer than 200,000 people in the U.S. CF affects 40,000 Americans and 105,000 people worldwide, classifying it as a rare disease. Although individually these diseases are “rare,” collectively they are far from it. Rare diseases impact over 30 million people in the U.S. and 400 million worldwide—yet 90% still lack an FDA-approved treatment. The numbers tell a staggering story: the rare disease community is vast, yet too often, they are left to fight for attention, funding, and research. For those with CF, this reality is especially stark.

Det sällsyntaste av det sällsynta

While CF is a rare disease in its own right, what many people do not realize is there is a rare subpopulation within the CF community—those in the final 10% that do not benefit from currently available mutation-targeted therapies. While groundbreaking mutation-targeted therapies have revolutionized life for 90% of the CF population, people like our co-founder, Emily, are part of the outlying 10%. They have rare genetic mutations of CF that make them the rarest of the rare. They are waiting for their lifesaving treatment as they have watched the rest of the CF community get their second lease on life. It’s an incredibly difficult place to be.

In addition, within the final 10%, there is a disproportionate prevalence of historically marginalized, nonwhite individuals with rare or understudied CF mutations. Among Hispanic, Black, Asian, and Native American CF populations, 17 to 40% of individuals do not benefit from existing mutation-targeted therapies. Ineligibility for mutation-targeted therapies contributes to existing health inequalities already facing these groups, including late diagnosis.

Charting a New Course

The 2025 #ExtraordinRARE campaign is more than a call for awareness—it’s a movement to accelerate action. Our theme this year is built around the idea of an expedition—an uncharted journey through the unknown, where every step forward brings us closer to the breakthroughs we need. The visuals reflect this sense of adventure, with bold imagery that captures the pioneering spirit of our mission.

And we are not venturing into the unknown empty-handed. Armed with innovation, collaboration, and an unwavering commitment to progress, we are:

• Funding transformative research-–With 38 groundbreaking grants awarded, we are propelling CF science into new frontiers.
• Developing treatments for antibiotic-resistant infections—We have helped develop 37 phages, which are viruses that kill bacteria, to combat antibiotic-resistant infections, a solution with implications far beyond CF.
• Creating pathways to clinical trials—Our CF Clinical Trial Connect (CTC) and Clinical Trial Matchmaking Program ensure that those in the final 10% have a direct line to clinical trial opportunities.
• Advancing research through the pipeline—We have advanced five projects to clinical stage, meaning studies being done on people with CF, which is a critical step in translating scientific discoveries into real-world therapies.

The Power of the #ExtraordinRARE

Being part of the ultra-rare disease community means challenging the status quo and realizing the power of your voice and your advocacy. It means forging new paths in science, refusing to accept “too difficult” or “too rare” as an answer, and—now, more than ever—proving that rare disease research can unlock new possibilities for all of medicine.

As we embark on this expedition, we do so with urgency, purpose, and an unrelenting drive to change the future—not just for those with CF but for the entire rare disease community.

Join the Expedition

The road ahead won’t be easy, but one thing is clear: we are not waiting for breakthroughs—we are creating them. We invite you to stand with us, raise your voice, and be part of this transformative movement.

 

How you can take action:

• Follow our #ExtraordinRARE journey on EE social media: Facebook | Instagram | LinkedIn | X 
• Spread the word and share your own rare disease adventures using #ExtraordinRARE
• Support research and innovation by donating to EE 
• Advocate for policies that impact research. One important timely issue involves reversing NIH indirect cost cuts, which would halt research not only for rare disease but across the board.  Take 60 seconds and contact your representatives.

To those still waiting: We see you. We stand with you. And we will not stop until you have the treatments you deserve.

The expedition begins now. Let’s move forward—together.

 

 

THANK YOU TO OUR 2025 RARE DISEASE WEEK SPONSORS