Research & Programs

The funds raised by Emily’s Entourage drive high-impact research, information exchange, and collaboration to accelerate breakthroughs for people with nonsense mutations of Cystic Fibrosis. We strongly believe that fast breakthroughs require the active participation of all stakeholders, including scientists, clinicians, foundations, patients and families, and industry. Our efforts to advance progress reflect that core value.  

Research Grants
Emily’s Entourage provides grant funding for research studies on nonsense mutations of Cystic Fibrosis with a focus on W1282X. This work is particularly significant because although nonsense mutations are relatively uncommon in the CF population, they are the cause of approximately one third of all genetic diseases. Progress on nonsense mutations of Cystic Fibrosis can have far reaching implications for many patients suffering from many different genetic diseases.

Emily’s Entourage has awarded grants to the University of California San Francisco, McGill University, The Hospital for Sick Children, and University of Alabama at Birmingham.

Grant funding decisions and priorities are made by the Emily’s Entourage Scientific Advisory Board, which consists of internationally renowned researchers, drug developers, and clinicians. This group vets, approves, and oversees research projects for Emily’s Entourage.

Scientific Symposia
Emily’s Entourage hosts scientific symposia. Previous symposia include a conference in partnership with the Penn Orphan Disease Center in January 2015 and a key opinion leaders (KOL) meeting in September 2015.

The first symposium brought the world’s top CF minds from biotech, academia, pharma and the CF Foundation together for the first time. The goal was to generate discussions, exchange ideas, and foster partnerships to expedite research and drug discovery and development for nonsense mutations using the W1282X mutation as a prototype.

The KOL meeting gathered world-class scientists and drug developers to brainstorm new, out-of-the-box approaches to treat Emily’s rare CF mutation and those like her, with a focus on therapies that could be developed quickly—within one to five years. Significantly, the summit included experts from inside and outside the CF community to facilitate cross-pollination of ideas and technologies.

Cell Donation
Emily has donated millions of her own nasal epithelial and iPs cells, which have been distributed to researchers and companies around the world to advance research and drug development for her nonsense mutation of CF.