“Perhaps the greatest contribution a physician can make to a rare disease field is not the successful treatment of a single patient but helping generate the evidence that transforms care for all patients.”
By any measure, this is a remarkable era for rare disease innovation. Advances in cell therapy, gene therapy, tissue engineering, and precision medicine have created opportunities that would have been unimaginable just a decade ago. Yet despite these scientific breakthroughs, many promising therapies face a challenge that has little to do with science.
