• Home
  • Emily’s Story
  • About Us
    • About Emily’s Entourage
    • Our Approach
    • Our Impact
      • 2022-2023 Impact Report
      • 2021 Impact Report
      • 2020 Impact Report
      • 2019 Impact Report
    • About Cystic Fibrosis
    • Meet Our Leadership
      • Scientific Advisory Board
      • Board of Directors
      • Leadership Team
    • Partner Organizations
  • Research
    • Areas of Focus
    • Awarded Grants
    • Funding Opportunities
      • Venture Philanthropy Investments
      • Collaborative Grants
      • Translational Grants
    • CF Clinical Trial Connect
    • Clinical Trial Matchmaking Program
    • The Final 10% Survey
    • Publications
    • For Researchers
      • Research Inquiries
      • Research Resources
  • Events
    • 2025 EE Gala
    • Events & Activities
  • Press & Media
    • EE in the News
    • Press Releases
    • Awards
    • Videos
  • Take action
    • Donate
    • Get Involved
    • Join CF Clinical Trial Connect
    • Shop EE
    • Sign Up for Updates
  • Blog
  • Contact
  • Donate
DONATE
  • Home
  • Emily’s Story
  • About Us
    • About Emily’s Entourage
    • Our Approach
    • Our Impact
      • 2022-2023 Impact Report
      • 2021 Impact Report
      • 2020 Impact Report
      • 2019 Impact Report
    • About Cystic Fibrosis
    • Meet Our Leadership
      • Scientific Advisory Board
      • Board of Directors
      • Leadership Team
    • Partner Organizations
  • Research
    • Areas of Focus
    • Awarded Grants
    • Funding Opportunities
      • Venture Philanthropy Investments
      • Collaborative Grants
      • Translational Grants
    • CF Clinical Trial Connect
    • Clinical Trial Matchmaking Program
    • The Final 10% Survey
    • Publications
    • For Researchers
      • Research Inquiries
      • Research Resources
  • Events
    • 2025 EE Gala
    • Events & Activities
  • Press & Media
    • EE in the News
    • Press Releases
    • Awards
    • Videos
  • Take action
    • Donate
    • Get Involved
    • Join CF Clinical Trial Connect
    • Shop EE
    • Sign Up for Updates
  • Blog
  • Contact
  • Donate

29 May 2019

Emily Speaks at Translate Bio’s CF Awareness Month Event

On May 29, Emily’s Entourage (EE) attended Translate Bio’s Cystic Fibrosis (CF) Awareness Month event in Boston. EE Co-Founder, Emily Kramer-Golinkoff, provided opening remarks to launch the day’s events as well as took part in a Q&A panel discussion.

On the panel, Emily was joined by key CF community members, Bob Coughlin, CEO of MassBio, Dr. Jonathan Zuckerman, a physician involved in Translate Bio’s ongoing clinical trial, and Dr. Ann Barbier, Translate Bio’s CMO. The panel was moderated by Dr. Meagan Vaughn, Senior Clinical Research Scientist at Rho, and delved into the urgent need for new treatment options in CF and how “someday isn’t good enough for the final 10%.” 

The session was livestreamed and can be viewed below. Members of the CF community were encouraged to tune in and sent in their questions.

During her remarks, Emily spoke about her own journey with nonsense mutations of CF as well as shed light on EE’s laser focused mission to develop life-saving treatments and drug development for the remaining 10%.

Following her remarks, Emily participated in a panel discussion covering topics such as the current state of CF treatment innovation, the potential role that emerging therapies may play in the future, and the burden that 10% of the CF community is still carrying.

Translate Bio is a mRNA therapeutics company currently developing medicines to treat diseases caused by protein or gene dysfunction, such as CF. With game-changing breakthroughs on the immediate horizon for roughly 90% of individuals with CF, those with nonsense mutations represent the majority of the outlying 10% who will not benefit from these breakthroughs.

Emily’s Entourage is incredibly honored to have participated alongside such a special lineup of key players in the CF community to raise awareness of the critical work that still needs to be done for the final 10% of the CF community. Huge thank you to Translate Bio for organizing such a wonderful event and for including EE and Emily in it!

boston, cf awareness month, cystic fibrosis, nonsense mutations, rare disease, translate bio
Share Post: facebook-share linked-share twitter-share
Appreciate Every Single Smile: Navigating Life with CF
Address the Unseen Scars: Mental Health and Cystic Fibrosis
The opinions stated by our guest contributors reflect the experiences and thoughts of the author alone. Our publication of these pieces does not reflect Emily’s Entourage’s endorsement or suggest a position on those opinions. The content on this site is for informational or educational purposes only and does not substitute professional medical advice or consultations with healthcare professionals.
  • Emily's Entourage Nonprofit Overview and Reviews on GreatNonprofits
    Volunteer. Donate. Review.
  • Home
  • About Emily’s Entourage
  • Careers
  • Contact Us
  • Donate
Sign Up for Updates >>

Emily’s Entourage
PO Box 71
Merion Station, PA 19066

Tax ID #45-3768161

Emily's Entourage
Website Managed By: Strategic Websites
  • Home
  • Emily’s Story
  • About Us
    ▼
    • About Emily’s Entourage
    • Our Approach
    • Our Impact
      ▼
      • 2022-2023 Impact Report
      • 2021 Impact Report
      • 2020 Impact Report
      • 2019 Impact Report
    • About Cystic Fibrosis
    • Meet Our Leadership
      ▼
      • Scientific Advisory Board
      • Board of Directors
      • Leadership Team
    • Partner Organizations
  • Research
    ▼
    • Areas of Focus
    • Awarded Grants
    • Funding Opportunities
      ▼
      • Venture Philanthropy Investments
      • Collaborative Grants
      • Translational Grants
    • CF Clinical Trial Connect
    • Clinical Trial Matchmaking Program
    • The Final 10% Survey
    • Publications
    • For Researchers
      ▼
      • Research Inquiries
      • Research Resources
  • Events
    ▼
    • 2025 EE Gala
    • Events & Activities
  • Press & Media
    ▼
    • EE in the News
    • Press Releases
    • Awards
    • Videos
  • Take action
    ▼
    • Donate
    • Get Involved
    • Join CF Clinical Trial Connect
    • Shop EE
    • Sign Up for Updates
  • Blog
  • Contact
  • Donate
English
Español Português Português do Brasil עִבְרִית Français Français du Canada Italiano Türkçe Svenska Deutsch