Cell & Gene Unscripted: Rare And Genetic Diseases

Millions of people around the world live with a rare disease. Many of them, along with their families, wait years for a diagnosis, have few treatments to choose from, and run into obstacles when they try to get care. Cell and gene therapy is moving quickly, though, and it's opening new options for conditions that have long had little or nothing to offer patients.

On this episode of Cell & Gene Unscripted, Gvantsa Kvantaliani, Associate Medical Director, Rare and Genetic Diseases, talks about the rare disease community from several angles at once: as a physician, a rare disease specialist, a patient advocate, and the parent of a child with a rare genetic condition. She speaks candidly about what families go through as they search for a diagnosis and then for treatment. She also makes the case for diagnosing patients earlier, for strong advocacy, and for better coordination among researchers, industry, healthcare systems, and government so more patients can get care.