A new clinical trial designed in collaboration between NTAP, academic, and industry partners aims to change the outlook for people living with cNF. The study is testing whether mirdametinib, a targeted therapy recently approved by the FDA for plexiform neurofibromas, can also offer relief from the debilitating burden of cutaneous tumors in adults.

Promising therapies are being developed to treat Neurofibromatosis (NF). These treatments are tested through clinical trials, which are only open to people who meet certain requirements. Each trial has its own specific criteria, which will be different for each trial.  By joining a clinical trial, people with NF can play an important role in helping researchers find better treatments and, eventually, a cure.

We are excited to host Dr. Donald Bearden, who will present on "The Brains Behind the Diagnosis: Cognitive and Emotional Development in Children with NF1." He will address the challenges faced by children with neurofibromatosis type 1 (NF1) beyond the physical manifestations, shedding light on cognitive deficits and emotional difficulties.

Britney’s first vivid memory related to her health occurred when she was only 5 years old and had her first MRI. Following her diagnosis, she became a frequent visitor to various doctors and medical students eager to learn about the girl with NF1. “I remember being told I wouldn't be able to play sports,” Britney shared. However, the spirit of defiance took hold during a car ride home when she expressed her desire to play soccer. Her parents agreed, and to everyone’s surprise, she excelled.