Myhre Syndrome Foundation Research Roadmap

The Myhre Research Roadmap is a five-year plan created by and for families and individuals affected by Myhre syndrome. It outlines our goals to drive research forward with speed and precision. We’re proud to build on the solid foundation of our first roadmap and move into a new accelerated phase of the MSF Research Pipeline. To do this, we’ve shifted from a step-by-step roadmap to a research pipeline. Think of it like a well-organized assembly line: different parts of research happen simultaneously, but they all connect to move ideas from the lab to treatments more efficiently.

MSF research pipeline: The research projects move from one domain to the next but are developed simultaneously in parallel. PPI: Protein-Protein Interaction. NCE: New Chemical Entity. FAP-CAR-T: Fibroblast Activation Protein Chimeric Antigen. Receptor T cells therapy. KI: Knock In. PK/PD: Pharmacodynamics/Pharmacokinetics. LNHS: Longitudinal Natural History Study.