MedCity News May 22, 2024
To win the battle against rare diseases, drug developers must work hand-in-hand with patients.
My colleagues and I were privileged earlier this year to meet with a caregiver and a young patient living with a rare form of pediatric cancer, who shared their stories and inspiring words of encouragement for the biopharmaceutical industry. Their message resonated with all who were there: Together we are working toward a world where every cure is possible.
It’s a big vision. Every day, more than 400 million people around the world are battling a rare disease. Many of those people are facing a scarcity of treatment options, often limited to drug regimens that haven’t been updated in decades and that do little to cure...
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