This weekend, an advisory board meeting was held by Agios Pharmaceuticals and Cambridge Biomarketing in Austin, Texas. It was the occasion for patients having pyruvate kinase deficiency to meet fellow patients or parents and share some knowledge about living with this rare disease.
This is a short description of the weekend by one of the patients:
“On the first day, we met with other pyruvate kinase deficiency patients and parents at the hotel. We had dinner together and were joined by Agios and CB.
The second day, Agios made a presentation about the preparations to start a phase 2 clinical trial of a medicine for pyruvate kinase deficiency patients. Then, each patient or parent made a timeline of important life events en presentations were prepared in groups. The day ended with some sightseeing.
The third and last day the presentations were given. There was a lot of discussion between presenations and this was an ideal occasion for sharing between fellow patients and parents. Some of us even got over their fear for public speaking!
The whole weekend was a very emotional and unforgettable experience.”