Emily's Entourage

Emily's Entourage is a nonprofit organization that raises money and awareness to help find a cure for rare ("nonsense") mutations of cystic fibrosis (CF), a genetic disorder that generally affects a person's lungs and digestive system. The foundation has raised over $1 million, largely from viral fundraising through universities and video campaigns. The organization also participates in exploratory research initiatives aimed at understanding and advancing towards a cure.

Emily's Entourage
FounderEmily Kramer-Golinkoff
Registration no.45-3768161
PurposeAccelerating cystic fibrosis research to save lives through the power of community, with a focus on "nonsense" mutations
Location
Websiteemilysentourage.org

Emily's Entourage is named after Emily Kramer-Golinkoff, a woman with CF, who has been the focus of an increased trend of patients participating deeply in research on and advocacy for their own conditions. She was honored at the White House as a Champion of Change for "harnessing data to take account individual differences in people's genes, environments, and lifestyles into account to improve patients' health."

Personal backgroundEdit

Kramer-Golinkoff was born on January 9, 1985. At six weeks old, she was diagnosed with CF.[1][2] At a young age, her mother, Liza, would pound "on her chest for 45 minutes to loosen the mucus in her lungs."[3]

Today, her lungs function at about one third of the normal lung function.[4][5] Emily undergoes a daily regimen of three to four hours of "airway clearance and breathing treatments" in addition to taking approximately 30 pills and four shots of insulin.[3][6]

Kramer-Golinkoff has a rare form of CF with a high prevalence among Ashkenazi Jews.[3][7] Her mutation belongs to a larger category called nonsense mutations, which affect approximately 10% of CF cases worldwide and account for up to 30% genetic diseases more broadly.[8]

She graduated in 2003 from Lower Merion High School in Ardmore, Pennsylvania.[9][10] She graduated cum laude from the University of Pennsylvania's Annenberg School for Communication and then completed her master's degree in bioethics at the University of Pennsylvania Perelman School of Medicine in 2009.[10]

Emily's EntourageEdit

Emily's Entourage was founded by Kramer-Golinkoff, her friends, and her family in December 2011 when Kramer-Golinkoff and her family sent a video to their friends and family and it raised over $40,000 in one week.[1][11] College campuses around the country have joined the movement by forming official clubs on campus and throwing annual fundraising benefits.[12][13][14][15] Local groups, such as Lower Merion School District often volunteer with Emily's Entourage.[9]

By May 2015, Emily's Entourage had raised over $1 million.[16][17]

ResearchEdit

Emily's Entourage has a Scientific Advisory Board to "set research priorities and vet, approve, and oversee research projects" for the organization.[18] In January 2014 Emily's Entourage hosted a research symposium on nonsense mutations in CF with a focus on Kramer-Golinkoff's particular rare mutation with leading researchers from biotech, academia, pharma and the Cystic Fibrosis Foundation in partnership with the Penn Orphan Disease Center to spur discovery of new treatments for those with nonsense CF mutations.[19][20]

Kramer-Golinkoff also published "A Lesson in Participatory Research for a Rare Mutation of Cystic Fibrosis" in the Journal of General Internal Medicine.[21] For her work towards finding new cures, she was honored at the White House as a "Champion of Change".[22][23][24][25][26] The honor focused on "work being done by patients, researchers, innovators, and advocates who are advancing our understanding of health and disease by harnessing data to take account individual differences in people's genes, environments, and lifestyles into account to improve patients' health."[27]

An NF1 clinical trial is undergoing FDA approval. Kramer-Golinkoff said, “I realized I could wait on the sidelines and pray for a miracle or get into the game and try to make one.”[28]

MediaEdit

Kramer-Golinkoff and Emily's Entourage have received significant regional and national media coverage. The attention has driven donations as well research opportunities and public awareness. Philadelphia Magazine profiled Emily as a "Health Hero" in September 2015. Kramer-Golinkoff said, "My biggest motivations for working my hardest to stay as healthy as possible — even in the face of an advanced and progressing fatal illness — are my family and friends, this burning desire to do more things and see more places, and the unbelievable Entourage that rallied behind me to give me and so many others real, tangible hope for that chance."[29] People magazine wrote, "Perhaps the most critical of Emily's Entourage's efforts has been its success in putting a beautiful face on an ugly issue. Case in point: The test tubes that fill the testing laboratories at USCF are all marked with random alphanumerical codes – except those marked with Emily's name."[30]

In Philadelphia Style magazine Kramer-Golinkoff emphasized her organization's personal approach: "We're trying to use my story to humanize the disease... People really respond to faces and stories and families in a way that's different. There are so many important causes out there, but we can touch people's hearts by making them feel like I could be anyone's daughter or sister or best friend.'"[31] Yahoo! Health profiled Kramer-Golinkoff. She said, "My disease is progressing and our race is getting more urgent. We're doing some really groundbreaking work that is changing the paradigm of how research is done, what gets attention, who the players are, and what the pace of progress is. We are constantly pushing the envelope because I literally don't have time to wait."[32]

CNN also profiled Kramer-Golinkoff. University of Pennsylvania Physiology professor Kevin Foskett said in the article, "Emily and her family want research focused on her mutation to be as focused and accelerated as possible. At one level, that sounds a little selfish, until you realize that her type of mutation, a 'nonsense' mutation ... is responsible for disease in many other genetically inherited forms of disease... Emily's story is compelling. If you've met Emily... you cant help but become engaged and energized." Kramer-Golinkoff said in that piece, "Obviously our prayer and dream is to have a breakthrough. But we also realize that it might not happen, or it might not happen in time for me. And I think that in the worst case scenario, even if it never could help me, there is a peace I get from at least knowing I'm doing everything I can, and it's not for lack of trying."[33]

See alsoEdit

ReferencesEdit

  1. ^ a b "Meet The Woman Behind Emily's Entourage". Fox 29. June 5, 2013. Archived from the original on March 27, 2015. Retrieved April 17, 2015.
  2. ^ "Emily's Story". Emily's Entourage. 2011-10-14. Retrieved April 17, 2015.
  3. ^ a b c "A Matter of Life and Breath". The Pennsylvania Gazette. February 27, 2014. Retrieved April 17, 2015.
  4. ^ "The Insider: Students join Emily's Entourage". Tulane University. May 7, 2012. Archived from the original on March 17, 2015. Retrieved April 17, 2015.
  5. ^ Faske, Brandon (April 21, 2014). "Emily's Entourage: College Students Rally to Cure Rare Form of Cystic Fibrosis". The Huffington Post. Retrieved April 17, 2015.
  6. ^ Mcmanus, Diane (December 12, 2012). "Breathing Life Into the Battle with Cystic Fibrosis". The Jewish Exponent. Retrieved April 17, 2015.
  7. ^ Elkin, Michael (January 29, 2015). "Emily's Entourage Shows Up in Force". The Jewish Exponent. Retrieved April 17, 2015.
  8. ^ Wilschanski, M (2012). "Class 1 CF Mutations". Frontiers in Pharmacology. 3: 117. doi:10.3389/fphar.2012.00117. PMC 3379685. PMID 22723780.
  9. ^ a b "LMSD students join "Emily's Entourage" to raise funds, support alumna". Lower Merion High School. December 4, 2012. Archived from the original on June 15, 2015. Retrieved April 17, 2015.
  10. ^ a b Fram, Joel (November 29, 2012). "Main Line volunteer of the week". Main Line Media News. Retrieved April 17, 2015.
  11. ^ Mimidis, Courtney (December 9, 2011). "Local Girl With Cystic Fibrosis is Making a Difference". WCAU. Retrieved April 17, 2015.
  12. ^ "HuffPost Live". huffingtonpost.com. Retrieved 11 June 2015.
  13. ^ "Entourage Raises Money For Cystic Fibrosis". WDSU. 15 May 2012. Retrieved 11 June 2015.
  14. ^ Davenport, Claire (March 13, 2015). "Tulane keeps up the fight against cystic fibrosis". Tulane University. Archived from the original on March 17, 2015. Retrieved April 17, 2015.
  15. ^ Kramer-Golinkoff, Emily (March 7, 2013). "How To Be An Empowered Patient: Emily's Entourage". Retrieved April 17, 2015.
  16. ^ Boccella, Kathy (February 28, 2014). "Emily's Entourage seeks cure for rare form of cystic fibrosis". Philly.com. Retrieved April 17, 2015.
  17. ^ "Emilys Entourage » Make Some Noise: May is Cystic Fibrosis Awareness Month!". emilysentourage.org. 2015-04-30. Retrieved 11 June 2015.
  18. ^ "Scientific Advisory Board". 2014-08-20. Retrieved April 17, 2015.
  19. ^ "Focus on Rare Diseases is Common at Penn Medicine - Penn Medicine News Blog". pennmedicine.org.
  20. ^ "Emilys Entourage » Emily's Entourage Symposium on Nonsense Cystic Fibrosis Mutations". emilysentourage.org. 2015-01-28.
  21. ^ Kramer-Golinkoff, Emily (July 17, 2014). "A Lesson in Participatory Research for a Rare Mutation of Cystic Fibrosis". Journal of General Internal Medicine. 29 (3): 808–809. doi:10.1007/s11606-014-2923-9. ISSN 0884-8734. PMC 4124119. PMID 25029983.
  22. ^ "The White House Champions of Change Program". whitehouse.gov. Archived from the original on 2017-01-20. Retrieved 2015-07-13 – via National Archives.
  23. ^ "Emily's Entourage: Giving Hope to Orphans of Orphan Disease". whitehouse.gov. 2015-07-09. Retrieved 2015-07-13 – via National Archives.
  24. ^ "White House honors 9 as precision medicine champions". MedCity News. 2015-07-06. Retrieved 2015-07-13.
  25. ^ "Local Woman Named White House 'Champion Of Change'". 2015-07-08. Retrieved 2015-07-13.
  26. ^ "Montco woman to be honored by White House". PhillyVoice. Retrieved 2015-07-13.
  27. ^ "White House Champions of Change - Precision Medicine". Youtube. The White House. July 8, 2015. Retrieved July 13, 2015.
  28. ^ "The Art of Medicine: Synergy Leads to Healing, at Medicine X | Ed". www.mysciencework.com. Retrieved 2015-12-08.
  29. ^ "Meet a Health Hero: Emily Kramer-Golinkoff". Philadelphia Magazine. 14 September 2015. Retrieved 2015-12-08.
  30. ^ "30-Year-Old Woman Races Time to Find Cure for Rare Form of Cystic Fibrosis: It's 'Just Outside of Our Grasp'". PEOPLE.com. Retrieved 2016-02-04.
  31. ^ "Why This Main Line Nonprofit Is Getting Support from the White House". phillystylemag.com. Retrieved 2016-02-04.
  32. ^ "She's 31 and Has Advanced Cystic Fibrosis — But Emily (and Her 'Entourage') Won't Give Up". www.yahoo.com. Retrieved 2016-02-04.
  33. ^ Chris Welch (8 April 2016). "Cystic fibrosis research: Emily's Entourage raises millions". CNN. Retrieved 2016-05-09.