To date, there is no cure for NF1 and there are no effective therapies to reverse disease symptoms or to prevent new ones from arising. Research and development in the field is hampered by several challenges, including:
* A highly variable clinical care setting
* Limited access to patient tissue samples for clinical research
* Lack of biomarker discovery and development efforts
* Limited understanding of the underlying disease biology
FasterCures, a center in the The Milken Institute, has convened 35 world-renowned experts from the neurofibromatosis (NF) research community, various biomedical disciplines, patient foundations, the pharmaceutical industry, and the National Institutes of Health to discuss the state-of-the-science of NF and the barriers to research progress.
The Milken Institute Philanthropy Advisory Service developed a Giving Smarter Guide for NF1 to help patients, supporters, and other stakeholders understand the state of the science and make informed decisions when directing their philanthropic investments. Readers will be able to use this guide to pinpoint research solutions aligned with their interests. This guide will help answer the following questions:
* What are key facts about NF1?
* What is the current state of care?
* What is the current state of research?
* What are major barriers to progress?
* Why should I invest in NF1 research?
* How can philanthropy advance new therapies for NF1?