Delivering on the Vision of Bench to Bedside: A Rare Disease Funding Community Collaboration to Develop Effective Therapies for Neurofibromatosis Type 1 Tumors

Abstract

AbstractThe time from target identification to new drug approval is often measured in decades. This can be even more challenging for rare diseases. Indeed, 95% of rare diseases do not have a specific therapy approved. Coordinated efforts to support research along the drug development pipeline can provide long term and comprehensive support to enable scientific breakthroughs for rare diseases. However, this requires coordination across multiple stakeholders. The present article analyzes the funding efforts of four major federal and philanthropic organizations to accelerate the advancement of MEK inhibitors to human clinical trials for NF1-associated tumors.