Congratulations, Glenn and Dr. Cara O'Neill! The FDA has approved Fayuvi, the first-ever treatment for Sanfilippo syndrome type A. This milestone is also a reminder of what patient-led advocacy and gene therapy can do for rare disease communities, including ours.

NF2 impacts entire families, and for Lizzie and Alanna, the journey has included surgeries, scans, specialist visits, and constant resilience. Behind every appointment and every procedure are real people fighting to hold onto their health and their futures.

Every NF2 diagnosis involves a child facing extraordinary challenges. A.J. was diagnosed at 6 and continues to manage multiple tumors and ongoing monitoring, but his strength shines through daily.

Lyren passed away in 2019, but her courage, strength, and spirit continue to inspire our community and our mission to find a cure for NF2. Her story is a reminder that behind every diagnosis is a person deeply loved by family and friends, and why this fight matters so much.

Behind every diagnosis is a person. A family. A fight. NF2 is not just a condition: it’s a daily reality for children, parents, and loved ones navigating uncertainty, treatment, and hope. Their stories are why we exist. Their strength is why we keep going.

Behind every NF2 diagnosis is a story, and a child, a family, a future. Tucker’s journey began with small changes that led to a life-changing diagnosis. Stories like his remind us why this fight matters so much. Help us turn stories like Tucker’s into hope.

NF2 is rare, but it affects real people in every community. Most are diagnosed young. There is still no approved treatment. But there is hope. Gene therapy research is advancing and with your support, we can help move these breakthroughs closer to real treatments for patients.

Before a therapy can be tested in people, it must first be shown to be safe, high-quality, and ready. That’s the work happening now, and it’s what your support makes possible. Closer to clinical trials. Closer to a cure. Donate today and be part of this progress: