EE Launches “Raring to Go!” Campaign for Rare Disease Week

Rare Disease Week, an annual, worldwide campaign to raise critical awareness for rare diseases, launches today in celebration of Rare Disease Day on February 29, 2024. This week is a special — and important — time for the rare disease community to unite and demonstrate collective support to raise vital awareness and ultimately drive more research to improve the lives of those affected by rare diseases, including cystic fibrosis (CF) and rare mutations of CF — a rare group within a rare disease!

At EE, we are laser-focused on the final 10% of the CF community with rare and nonsense mutations, and we are so proud to be part of the incredible rare disease community. We have witnessed time and time again the power, strength, and determination of the rare disease community, and for that reason — and many others — we are excited to participate in and support Rare Disease Week with the launch of our “Raring to Go” campaign!

What is a Rare Disease?

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, qualifying it as a rare disease. Although individually these diseases are “rare,” collectively they are far from it. There are more than 7,000 rare diseases affecting more than 30 million people in the US — or roughly one in ten Americans. Think of it this way: that is one person on every elevator and four people on every bus. While we call these diseases rare, in reality, they are quite prevalent, and sadly, there are currently no approved treatments for more than 90% of these rare diseases. We have our work cut out for us! 

The Rarest of the Rare

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. 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. During Rare Disease Week, we are featuring powerful stories that highlight these experiences and bringing critical awareness to the unmet needs of these underserved CF populations.

“Far too often, those in the rare disease community, including those from nonwhite, historically marginalized groups, are overlooked, marginalized, made to feel too rare to matter. At Emily’s Entourage, we are steadfast in our belief that every single human life matters, infinitely, no matter how rare your mutation or disease may be,” said EE’s co-founder Emily Kramer-Golinkoff.

Through our Rare Disease Week campaign and our relentless advocacy for the CF community, our goal at EE is to raise awareness and expedite critical research and drug development advancements for the 100% of the CF community so nobody is left behind.

Introducing “Raring to Go!”

To recognize Rare Disease Week 2024, today EE launches a week-long digital campaign, “Raring to Go,” to highlight those living with rare mutations of CF and the broader rare disease community, raise awareness of the challenges they face, and bring critical attention to their urgent, unmet need for lifesaving therapies

Why “Raring to Go”? Because with a fatal illness like CF, days matter. We must move FAST to advance lifesaving therapies for those who desperately need them and whose lives depend on them. Speed is the name of the game! And so we charge forward at turbospeed to drive accelerated progress for the final 10% of people with CF. Vrroooom!

Throughout Rare Disease Week, EE’s “Raring to Go” campaign will feature inspiring stories from the CF community and facts about rare disease. This week — and always — we encourage everyone to celebrate the rare disease community and their rare, extraordinary strength, resilience, and courage. We invite you to join us in our urgent race to speed the development of lifesaving therapies so they no longer have to be so supernaturally strong and extraordinary.

Are you “Raring to Go”?

Let’s do this! We hope that this Rare Disease Week, you will feel energized to join in and help us recognize and raise awareness for the exceptional rare disease community. Head to our social media channels — Facebook, Instagram, X, and LinkedIn — to join the conversation!

Thank You to Our Rare Disease Week Sponsors

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