Overall Objective:

1. Describe the development of a rare disease registry;
2. Discuss how rare disease registries inform clinical trials;
3. Discuss ways to optimize the approach to monitoring in patients with congenital hemolytic anemias.

Faculty/Planning Committee:

Rachael Grace, MD, Speaker, is an consultant for Adios and Principia, and received grant research support from Novartis and Dova.
Faculty Member does plan to discuss investigational uses of a commercial product: Gene therapy for pyruvate kinase deficiency, mitapivat for sickle cell anemia and thalassemia.

Disclosure Policy:
Boston University School of Medicine asks all individuals involved in the development and presentation of Accredited Continuing Education activities to disclose all financial relationships with ineligible companies. This information is disclosed to all activity participants prior to the start of the educational activity. Boston University School of Medicine has procedures to mitigate all relevant financial relationships with ineligible companies. In addition, faculty members are asked to disclose when any unapproved use of pharmaceuticals and devices is being discussed. In accordance with the Standards for Integrity and Independence in Accredited Continuing Education, all relevant financial relationships with ineligible companies that faculty, planners, authors and anyone who may be in control of content have been mitigated.