Research
Advancing science to find effective treatments and a cure for all forms of neurofibromatosis.
Pursuing Treatments and a Cure
Neurofibromatosis research has entered an exciting era. With the first FDA-approved treatment for NF1 now available and dozens of clinical trials underway, the path toward effective therapies for all forms of NF has never been clearer.
Clinical Trials
Over 60 active clinical trials are exploring targeted therapies, gene therapy, and immunotherapy approaches for NF1, NF2, and schwannomatosis.
Basic Science
Researchers are uncovering the molecular pathways that drive tumor growth, opening doors to precision medicine approaches tailored to each type of NF.
Drug Development
From MEK inhibitors to gene therapies, pharmaceutical and academic labs are developing next-generation treatments that target NF at its genetic roots.
Federal funding through the NIH and the Congressionally Directed Medical Research Program (CDMRP) has been the backbone of NF research for decades. The NF Network works year-round to protect and grow this critical investment — because every dollar brings us closer to a cure.
Your Role in Advancing Science
Patients and families are at the heart of NF research. By participating in clinical trials, joining patient registries, and sharing your experience, you help drive the discoveries that lead to better treatments.
How to Get Started
Learn About Trials
Browse clinicaltrials.gov or speak with your NF specialist to understand which studies may be a good fit for you.
Talk to Your Doctor
Discuss participation with your healthcare team. They can help you weigh the benefits and risks of any study.
Connect with the NF Network
Our team can help you navigate the research landscape, connect with study coordinators, and answer your questions.
Stay Engaged
Even after a trial ends, your involvement matters. Share your experience, join a registry, and help spread the word.
Research Milestones
The NF research community has achieved remarkable progress in recent years. These breakthroughs are transforming what it means to live with neurofibromatosis.
FDA Approves Selumetinib (Koselugo)
The first-ever FDA-approved drug for NF1, selumetinib (a MEK inhibitor) was shown to shrink plexiform neurofibromas in children, marking a historic milestone for the NF community.
NF2 Gene Therapy Advances
Early-stage clinical trials for gene therapy targeting NF2-related vestibular schwannomas have shown promise, offering new hope for preserving hearing in NF2 patients.
Biomarker Discovery for Schwannomatosis
Researchers identified novel biomarkers that may help diagnose schwannomatosis earlier and track disease progression, paving the way for more targeted clinical trials.
AI-Powered Imaging for NF Tumors
Machine learning tools were developed to automatically segment and measure NF-related tumors on MRI scans, improving the accuracy and speed of clinical trial assessments.