The Global Genes RARE Advocacy Summit in September 2023 provided a forum for me to talk, for the first time, about my upcoming book and how rare disease communities have been a key part of my professional life. Here’s a set of questions that Sravanthi Meka of Global Genes asked me in advance of the meeting: […]
Solvers
Wow! How? Patient-Led Research
A scientific journal article written primarily by patients about their health condition has been downloaded over a million times. We reached this milestone after years of activism, advocacy, and demands for justice. The internet supercharged everyone’s ability to connect with information and with each other. The crucible of the pandemic then forced us into new […]
Wow! How? Gleevec
When Norman Scherzer’s wife, Anita, was diagnosed with a rare form of cancer in 1994, he found that the most up-to-date information was being traded among patients who were connecting via email. When a new test, a new drug, and a new clinical trial were all developed at the same time, Scherzer helped organize the […]
Wow! How? End of life
What if I told you that a key health care metric grew in a positive direction, from 24% to 31% in 15 years? What if I then told you that a big part of the change was driven by people inspired by personal experiences, not clinical training? Most Americans tell survey researchers they don’t want […]
Wow! How? False Boundaries
This is a cross-post from my LinkedIn newsletter. Feel free to join the conversation there or post your thoughts in the comments below. Every single day, Emily Kramer-Golinkoff takes 30+ pills, plus insulin, and engages in hours of breathing treatments. She is a sought-after expert on patient-driven scientific discovery, having co-founded a nonprofit, Emily’s Entourage, that pursues breakthrough therapies for […]
Wow! How? Home inventors
Hot springs have always been therapeutic destinations, but it was a father, desperate to relieve his child’s pain, who invented the home spa. Candido Jacuzzi, a mechanical engineer, and his wife, Inez, tried everything to help their son, who was suffering from juvenile rheumatoid arthritis. Hydrotherapy helped immensely, so Candido set to work, eventually building […]
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