Tag: platform trials for rare diseases

  • Rare Disease Research Opens New Paths to Treatment

    Rare Disease Research Opens New Paths to Treatment

    A rare disease may affect relatively few people, but more than 7,000 such conditions together affect hundreds of millions worldwide. In this program, Researcher Stephanie Cherqui, Ph.D., explains why studying them matters not only to families facing a devastating diagnosis, but also to the future of medicine. Her work on cystinosis uses a patient’s own…