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Introducing Emily’s Entourage
Who is Emily?
Emily was born on 9 January 1985, and diagnosed with CF at six weeks old. A CF diagnosis is a shock for all parents, but Emily’s parents were determined to make her life joyous and fulfilling while ensuring she got the best medical care.
Now 40 years old, Emily has no day off, spending hours a day on breathing treatments, injecting insulin, and taking over 30 pills a day, as she is unable to benefit from modulators.
Emily’s story has touched thousands of people, and she was named a Champion of Change for President Obama’s Precision Medicine Initiative in 2015, is the recipient of the 2020 Philadelphia Magazine Luminary Award, and the 2016 Global Genes Rare Champion of Hope for Advocacy Award. Most recently she received the 2024 Leadership in Personalised Medicine Award by the Personalised Medicine Coalition.
At Emily’s Entourage, we believe that every single person with CF deserves a lifesaving breakthrough, no matter how rare their mutation.
Emily Kramer-Golinkoff
That's why we’re thrilled to partner with Cystic Fibrosis Trust to expand access to our CF Clinical Trial Connect database to people that don’t benefit from existing CFTR modulators in the UK and around the world. The more people we have in the CTC, the faster we can enroll trials and develop new therapies so no one with CF is left behind, and everyone with CF has the chance to live healthy, long lives.
What is Emily’s Entourage?
Emily’s Entourage (EE) started in Emily’s parents’ living room, but has now awarded millions of dollars in research grants, launched a now clinical-stage CF gene therapy company, developed a global patient database and clinical trial matchmaking program to accelerate clinical trial recruitment for people unable to benefit from modulators, and leads worldwide efforts to drive high-impact research and drug development for those that do not benefit from existing CFTR modulators.
Emily’s Entourage also conducts the Final 10% Survey to better understand the perspectives, unmet needs, and clinical research preferences of people with CF who aren’t able to benefit from or access modulators.
How does Cystic Fibrosis Trust work with Emily’s Entourage?
Alongside our other work through the Clinical Trials Accelerator Platform (CTAP), we’ve partnered with Emily’s Entourage so that people in the UK who are not able to benefit from current modulators can join their global patient database, CF Clinical Trial Connect (CTC).
The CTC is the only global patient database designed exclusively for those unable to benefit from modulators and aims to make it easier to connect people with relevant trial opportunities. Registering for the CTC only takes a few minutes and offers alerts to the latest relevant clinical trial opportunities — delivered directly by email. More information about how to join and be alerted of relevant UK trial opportunities can be found here.
You can also view trial opportunities on our Clinical Trials Accelerator Platform (CTAP), more information here.
We also have awarded a co-funded research grant with Emily’s Entourage to develop novel ways to deliver a genetic therapy for CF in the lab. In the future, genetic therapies could be a treatment option for everyone with CF including those who are unable to benefit from modulators, which is a top research priority for the CF community. More information about this project can be found here.
There are likely to be an increasing number of trials opening in the UK specifically for people who cannot currently take CFTR modulators. Alongside the other resources the Trust has created to support the community to learn about taking part in research – including our Trials Tracker – the Clinical Trial Connect database will help more people hear about clinical trial opportunities they may be eligible for.
Becca Brendell, Head of the Clinical Trials Accelerator Platform (CTAP)