A EE lança uma colaboração piloto de registo de doentes com a Translate Bio

In September, Emily’s Entourage (EE) launched a first-of-its-kind collaboration with Translate Bio to pilot its Cystic Fibrosis (CF) Nonsense Mutation Patient Registry as a tool for clinical trial patient identification and outreach. The goal is to connect individuals with CF with relevant clinical trial information and opportunities to speed the drug development process by making clinical trial recruitment more targeted and efficient, ultimately leading to faster development of life-saving therapies.

“We are thrilled to partner with EE and we believe the patient registry will be an important component of our MRT5005 enrollment strategy as we move forward in clinical development.”
~ Dr. Ann Barbier, Chief Medical Officer, Translate Bio

Emily and Liza touring the lab during a visit to Translate Bio in 2019.

“While there have been significant advances in the treatment of CF, substantial unmet need remains. Roughly 10 percent of the CF population is not anticipated to benefit from currently approved CFTR modulators,” said Dr. Ann Barbier, Chief Medical Officer, Translate Bio. “MRT5005, Translate Bio’s first clinical-stage mRNA product candidate, has the potential to treat all people with CF, including those with nonsense mutations. We are thrilled to partner with EE and we believe the patient registry will be an important component of our MRT5005 enrollment strategy as we move forward in clinical development.”

EE’s registry consists of individuals with one or two copies of a nonsense mutation of CF. It includes hundreds of individuals from 42 states and more than 33 countries around the world, ranging in age from 1 to 67 years old. The registry allows individuals with CF to learn about relevant research, clinical advances and drug trials, and to expedite the development of life-saving therapeutics.

Emily on a panel for Translate Bio’s CF Awareness Month Event in 2019

It is critical to note that the privacy of registrants is of utmost importance to EE. Individual personal information is never sold or shared with companies or any outside institutions.

EE is thrilled to expand the organization’s value to the CF community and biotech companies, and is particularly delighted to do so with Translate Bio!

Join EE’s CF Nonsense Mutation Patient registry >>

For inquiries or more information about partnering with the EE CF Nonsense Mutation Registry, please submit here >>