October 12, 2021

PA Rare Disease Organization Leader Profile: Emily Kramer-Golinkoff

This is the fourth in a series of profiles of the leaders of rare disease organizations based in Pennsylvania.

  1. Full Name: Emily Kramer-Golinkoff
  1. Current Hometown: Ardmore, PA
  1. Organization’s Name: Emily’s Entourage
  1. Mission of the Organization: Emily’s Entourage accelerates research and drug development for nonsense mutations of cystic fibrosis (CF). By providing critical leadership and coordination, Emily’s Entourage drives high-impact research, cultivates multi-stakeholder collaboration, and facilitates information exchange to speed breakthroughs. Our goal is singular: to expedite life-saving treatments and a cure for those with nonsense mutations of CF who are waiting with bated and fading breath.
  1. Rare Disease(s) Covered by the Organization: Cystic fibrosis – specifically nonsense mutations of cystic fibrosis
  1. Your Role at the Organization: Co-Founder
  1. How You Got Involved with the Organization: We started Emily’s Entourage because we saw a huge unmet need in the cystic fibrosis community. There are game-changing breakthroughs for roughly 90% of the CF community, but with 2 copies of a rare form of CF (called “a nonsense mutation”), I happened to fall in the outlying 10% that does not benefit from these revolutionary advances. To make matters worse, with a fatal disease like CF, we recognized that time was of the essence and we didn’t have time to wait for the traditional drug development process. So, we took matters into our own hands, launching Emily’s Entourage to speed research and drug development for nonsense mutations of Cystic Fibrosis.
  1. What You Are Most Proud of or What You Are Excited About Doing Next: I’m most proud that took a situation that was devastating and that easily could have been paralyzing, and we made something of it. We rejected the status quo, we defied the naysayers, we dreamt big and ran with it because we believed that the final 10% of the CF community that did not benefit from the game-changing targeted therapies, including those with nonsense mutations, deserved better and that time was of the essence. Since then, we’ve created real impact. It goes to show that you don’t have to be a billionaire or a biologist or a big time institution to make a real, measurable difference in the world. 
  1. What You Find Most Rewarding in Your Role: I’m incredibly energized by Emily Entourage’s entrepreneurial, innovative approach, and by the opportunity for direct impact. We are filling a critical gap, creating hope for the 10% of the CF community that has been left behind by the breakthroughs and we are working against the clock. 

    I see my friends with CF. I see how hard they work. I know how deeply they are loved and the important things they need to do in the world. And yet, I see and know all too intimately the horrors of CF. I see how despite our most herculean efforts, CF rages on, how it rips apart families, dreams, communities. I believe the CF community deserves better. 90% is remarkable, but it isn’t good enough. We can do better. That insatiable hunger for better solutions for 100% of the CF community is what drives me. I believe everyone with CF deserves realistic hope for a future, and it gives me tremendous purpose to be able to devote my life to helping to secure that for 100% of the CF community.

  1. What You Find Most Challenging in Your Role: Leading a nonprofit is hard enough as it is. Doing it with the advanced stages of the disease you are working so hard to create treatments and cures for makes it exponentially harder. I have 4-5 hours of daily medical treatments, medical appointments, health issues that pop up all the time, and all the other challenges that come with having the advanced stages of cystic fibrosis, 33% lung function, and CF-related diabetes. Balancing Emily’s Entourage and my own health needs is a constant, enormous challenge!
  1. Resources You Recommend to Other Rare Disease Advocates: We are very lucky to work closely with the Cystic Fibrosis Engagement Network, a consortium of many other CF advocacy organizations. CFEN and the CF advocacy organization community as a whole are incredibly collaborative and supportive. While we have different areas of specific focus, we all have an acute awareness of our shared end goal and that we are in this together. Nobody else has the same deep, vested interest in that end goal as we do. With that in mind, I’d encourage other rare disease advocates to build alliances with other organizations in your disease areas. Many times, those groups can be your greatest resources and most authentically aligned partners. 

    Beyond the CF community, I’ve been fortunate to find a group of mentors from other advocacy organizations who have been tremendous resources to me. They bring a wealth of experience, having faced many of the same challenges that we face as an organization and that I personally face as a leader. They’ve helped me navigate through daunting challenges and provided critical support, guidance, and resources every step of the way. I would encourage other advocates to build their tribe of mentors! They are the people who have “been there, done that” and can weigh in with a unique understanding of your situation and offer highly valuable and relevant expertise.

    There are also a number of other umbrella organizations that have been incredibly helpful for us, including the Michael Milken Institute’s FasterCures and Global Genes

  1. Any advice you’d like to share with the rare disease community in PA: Here’s a blog post I wrote a few years ago with specific lessons learned and suggestions >> https://www.emilysentourage.org/top-10-lessons-on-running-a-disruptive-foundation/
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