Cystic Fibrosis Podcast 192 Emily’s Entourage

In the latest Cystic Fibrosis Podcast, Jerry speaks with Emily Kramer-Golinkoff about the role of a patient advocate organization in driving drug development in rare disease.
Emily, a 33-year-old who has a nonsense mutation of CF, is a co-founder of Emily’s Entourage, a 501 3(c) that’s goal is to accelerate research for new treatments and a cure for CF. She is an internationally recognized patient advocate and speaker, has a Master’s degree in Bioethics and is certified in Clinical Ethics Mediation, was named “Champion of Change” by President Obama’s Precision Medicine Initiative, and has been featured by CNN.com, Time.com, AOL.com, People.com, and more for her work with her charity.

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Trial to Possibly Treat Nonesense Mutations Begins

Sevion Therapeutics and Eloxx Pharmaceuticals announced that a first healthy subject has been dosed in a Phase 1b clinical trial assessing the safety, tolerability and drug properties of ELX-02 as a potential treatment of several genetic diseases caused by nonsense mutations, including cystic fibrosis (CF).

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Biopharmaceutical company developing novel small molecule medicines for genetic diseases caused by nonsense mutations

http://markets.businessinsider.com/news/stocks/Eloxx-Pharmaceuticals-Announces-Expansion-of-Intellectual-Property-Estate-for-ELX-02-with-Newly-Granted-Patent-in-Europe-1004482616

Eloxx Pharmaceuticals Announces Expansion of Intellectual Property Estate for ELX-02 with Newly Granted Patent in Europe

Sevion Therapeutics, Inc. (OTCQB:SVON) and Eloxx Continue reading Biopharmaceutical company developing novel small molecule medicines for genetic diseases caused by nonsense mutations