Learn more about a new clinical trial for RUNX1-FPD patients which will evaluate the safety and tolerability of a medicine called sirolimus (also known as rapamycin).

Sirolimus is an immunosuppressant that can reduce inflammation by blocking a signaling pathway called mTORC1. It is being tested because scientists found that mTORC1 is elevated in the bone marrow of RUNX1-FPD patients.

Dr. Courtney DiNardo, the study’s principal investigator, provides an overview of the trial’s objectives, discusses the eligibility requirements for participation, and responds to questions from attendees. Dr. Anupriya Agarwal joined to share the lab research perspective from this study.