Bench to Bedside - Collaborating to Develop Effective Therapies for NF

Posted March 13, 2019

  Aaron Schindeler, Ph.D., The Children's Hospital at Westmead

“The 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 NFRP has been a critical partner in moving the field of NF research from understanding the basic biology of the disorder to identifying potential therapeutics and testing in clinical trials. A recent 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. Click Here to Read (External Link)

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Last updated Thursday, May 26, 2022