Building awareness and communities to support and find treatments for Rare Diseases and Genetic Conditions
A key theme of the World Orphan Drug Congress 2013 was the need to innovate when it comes to the rare disease space. Presenters shared how rare diseases necessitate a different approach, ranging from clinical trials to working with patients and the US Food and [...]
I attended the World Orphan Drug Congress last week in Washington, DC, and Siren Interactive presented the workshop, “A Roadmap for Patient Advocacy Investment.” In the rare disease space, patient engagement is critical to every step of a drug’s development, including filling clinical trials, getting [...]
I’ve met Julie Flygare, a passionate advocate for people with narcolepsy, who has a charming personality and is an engaging speaker. Last night I finished Julie’s book, Wide Awake and Dreaming: A Memoir of Narcolepsy, and learned that she is also an engrossing storyteller. Julie [...]
“Personalized medicine refers to the tailoring of medical treatment to the individual characteristics of each patient,” according to the President’s Council of Advisors on Science and Technology report, Priorities for Personalized Medicine. “It does not literally mean the creation of drugs or medical devices that [...]
As difficult as it is to have a rare disease, not having a diagnosis is even worse. We’ve blogged before about the fascinating work of the Undiagnosed Diseases Program (UDP) at the National Institutes of Health (NIH), whose goal is to provide answers to patients [...]
Have you ever wondered what your doctor is scribbling down while you are talking about all of your symptoms? Well, the “open notes” initiative wants you to be able to see those notes. It’s the idea that patients should have easy access to view the [...]
I was tweeting fast and furiously at the 7th Annual Rare Disease and Orphan Drug Leadership Congress on July 18 and 19 in Philadelphia, and here are some of the insights I captured. Stephanie Okey from Genzyme did an excellent job moderating and set the [...]
