The Rare Bone Disease Alliance is excited to announce the fourth Rare Bone Disease Alliance Scientific Symposium on November 21, 2024. In this series, researchers and expert speakers explore developments in rare bone diseases that are at various stages of treatment innovation and understanding of natural history. It is intended for researchers and clinicians interested in rare bone diseases. Developing therapies for rare bone diseases present unique challenges requiring multiple steps, from identifying cellular pathways and targets to conducting clinical trials in diverse and challenging patient populations.

Among rare diseases, the FOP research space is one of the most active and exciting. With the first-ever treatment for FOP approved in select countries and five active FOP clinical trials, there is still an abundance of preclinical research with the potential to be translated into safe and effective new medicines. Molecules advancing drug discovery, advanced therapeutic modalities and new technologies must continually be explored for their potential to treat understudied signs and symptoms of FOP.

In this “deep dive” into FOP, you’ll hear about an important N of 1 study known as The Resilient Patient Project that brought to light a potential new therapy for FOP which has recently been granted Orphan Drug Designation. You’ll also hear about a project that in the preclinical phase was funded in part by the International FOP Association and then progressed to a phase 2 clinical trial funded through a variety of innovative avenues.

Presentation titles and speakers will include:

Welcome Remarks
Eric Rush, MD, Children’s Mercy Hospital
Michelle Davis, IFOPA Executive Director

The Power of One
Frederick Kaplan, MD, University of Pennsylvania

Saracatinib: From the bench to a Phase 2 Investigator-led trial
Paul Yu, MD, PhD, Massachusetts General Hospital and Harvard Medical School

Funding an Investigator-led trial
Marelise (Elisabeth) Eekhoff, MD, PhD, Amsterdam University Medical Center

Closing Remarks
Eric Rush, MD, Children’s Mercy Hospital