During the BIO International Convention in June, we heard inspiring stories from patients and patient advocates about their experiences with rare disease diagnoses, the seemingly endless road to a cure or treatment, and the hope that our industry offers these patients in terms of a more healthy, happy life. We captured these stories on video – and they truly remind us all why we're in this industry.
Lauren Bombardier Weeks detailed her journey as a cystic fibrosis patient, which started 25 years ago when she got the diagnosis. Doctors were hopeful, but as the years went on, Weeks pictured her future as “a black expanse” that she’d never experience — and couldn’t even imagine.
“What do you do when 10 years of waiting turns into 20 years?” said Weeks. “I learned there was a good chance I wouldn’t make it into my 30s.”
Then, in August of 2015, Weeks stopped waiting and started living. ORKAMBI, the oral medication developed by Vertex Pharmaceuticals that had just been approved by the FDA, arrived on her doorstep.
“Not only did this drug open up the doors in my cells, but it opened up the door to my future,” said Weeks, who has since gotten married, earned her master’s degree and written a book. “My lung function, which kept going down, actually changed direction and increased. I was feeling better every day, and I knew my future was possible…. I still have cystic fibrosis, but it’s a less scary version of CF. I still remain hopeful for my future and the future of so many others because I’ve seen what is possible because of science.”
Hear directly from Lauren Bombardier Weeks:
If you're interested in hearing directly from patients like Weeks, and learning more about the incredible role of patient advocacy in drug development, register for MassBio's Patient Advocacy Summit on Nov. 2nd.