Research and the Diagnosis Journey

This episode follows bleeding disorders treatments from lab bench to the patient in the chair. On one end, the science: a pipeline that in the last decade has delivered gene therapy, factor-mimetic therapies, and rebalancing agents —treatments that have transformed how people with bleeding disorders live. On the other end, the reality of an HTC: what it takes to translate those advances into a patient's treatment plan, and what happens when insurance, geography, or missed diagnosis get in the way. Our two guests are hematologists experienced in both research and treating patients. Dr. Pipe and Dr. Kouides speak on the science and the standard. The goal is to make the diagnosis-to-treatment arc legible to listeners and to be honest about where the translation still breaks down.   The National Bleeding Disorders Foundation is dedicated to finding cures for inheritable blood and bleeding disorders and to addressing and preventing the complications of these disorders through research, education, and advocacy enabling people and families to thrive.  NBBF has information about current and future therapies on our website: Current therapies: https://www.bleeding.org/bleeding-disorders-a-z/treatment/current-treatments Future therapies: https://www.bleeding.org/bleeding-disorders-a-z/treatment/innovative-therapies Connect with NBDF at our website https://www.bleeding.org/ or follow us on Instagram https://www.instagram.com/nbd_foundation or on Facebook https://www.facebook.com/NationalBleedingDisordersFoundation