Driving breakthroughs for the NF community at the 2025 NF Conference

A new era is unfolding for the NF community. With two FDA-approved treatments now on the market - including the first-ever therapy for adults with NF1 - and cutting-edge advances in AI, drug repositioning, and precision medicine, momentum is building like never before. 

This progress takes center stage at the 2025 NF Conference, hosted by the Children's Tumor Foundation June 21–24 at the Omni Shoreham Hotel in Washington, D.C. As the global gathering point for neurofibromatosis and schwannomatosis (collectively known as NF), the Conference convenes the brightest minds in science, medicine, and industry - alongside the patients driving it all forward - to deliver faster answers and cures for over 4 million people living with NF. 

From gene therapy to regulatory reform, from platform clinical trials to next-generation pain treatments, the program dives deep into what's working, what's next, and how this moment can be a model for the rare disease field as a whole. This isn't just another scientific conference - it's a turning point. It's where science meets urgency, and where patient experiences drive scientific impact. 

NF is a group of genetic conditions that cause tumors to grow on nerves throughout the body and can lead to serious health issues like blindness, deafness, pain, and cancer. It affects 1 in 2,000 people of all backgrounds and includes NF1 and all forms of schwannomatosis, including NF2-related schwannomatosis (formerly referred to as NF2).

Kicking off the week is the 2025 NF Summit (June 19–21), where hundreds of patients, families, and NF advocates come together to share knowledge, build community, and lead change. The NF Summit is proof that when the NF community speaks with one voice, people listen. 

Keynote speakers: 

• Nancy Ratner, PhD, Progress and Future Prospects of Plexiform Neurofibroma Formation (Cincinnati Children's Hospital)

• David A. Gewirtz, PhD, Next generation treatments (Virginia Commonwealth University)

• William Gahl, MD, PhD, NIH, Managing diverse clinical manifestations and the NIH Undiagnosed Disease Program (National Human Genome Research Institute)

• Pelin Candarlioglu Deacon, PhD, Human-Centric Advanced Cell Models in Preclinical Drug Development (3D and 3Rs Ltd)

Highlights from the NF Conference Program:

• Young Investigator Day A full day dedicated to mentoring and supporting early-career NF researchers - fueling the next generation of innovation.

• AI and Next-Gen Therapies Explore how artificial intelligence, precision medicine, and next-gen platforms are transforming the treatment landscape.

• Global Regulatory Dialogue A unique panel featuring leaders from the FDA and the European Medicines Agency, on how regulation must evolve to meet the needs of rare disease patients.

• The Future of Pain This session spotlights how NF research is shaping new approaches to chronic pain - and what that means for broader innovation. 

• Friedrich von Recklinghausen Award Honoring Pierre Wolkenstein, MD, PhD, Henri-Mondor Hospital, Paris East University, for his significant contributions to NF care and research.