Offered by: Children’s Hospital of PhiladelphiaConducted by: Matthew Hocking, PhD The specific aims of this project are to describe parent’s perspectives on caring for youth with NF-OPGs, and the factors they consider in choosing neuroprotection, neuro-enhancement and vision restoration treatment strategies using focus groups of parents/LG of youth with NF-OPGs. We are looking to have…
Read MoreWhy Clinical Trials Matter, and Why They Matter Especially for NF
Every treatment that exists today: every medication, every therapy, every breakthrough, started somewhere. It started with a question, a hypothesis, and eventually, a clinical trial. May 20 is National Clinical Trials Day, a moment to recognize the remarkable system that turns scientific ideas into real-world answers. For the NF community (patients, families, caregivers, and advocates)…
Read MoreEvaluation of Acceptance and Commitment Training (ACT)
Remote Research Study: Support for Caregivers of Children with RASopathies Offered by: NIH/National Cancer Institute, Bethesda MDConducted by: Dr. Staci Martin Peron A remote research study from the National Cancer Institute is evaluating Acceptance and Commitment Training (ACT), a method that has been effective in helping parents of individuals with other chronic conditions. The study…
Read MoreStudy on Tumor Visibility
Offered by: NIH/National Cancer Institute, Bethesda MDConducted by: Dr. Staci Martin Peron You can help researchers better understand the experiences of people with NF1. The National Cancer Institute (NCI) is conducting a virtual study for individuals with visible plexiform or cutaneous neurofibromas, and you may be eligible to participate. About the Study The goal of…
Read MoreYour Letter Can Make a Difference for NF Research
We are collecting letters to hand-deliver to your members of Congress. Sharing your story helps emphasize the importance of continued NF research funding through the Congressionally Directed Medical Research Programs (CDMRP) and the National Institutes of Health (NIH).
Read MoreNF1 Treatment News: FDA Expands Selumetinib (KOSELUGO) Approval to Adults
NF Midwest is excited to share important news for our community, news that brings real hope to people living with Neurofibromatosis Type 1 (NF1). The FDA has officially approved selumetinib (KOSELUGO) for adults with NF1 who have symptomatic, inoperable plexiform neurofibromas (PN). While this MEK inhibitor has been available to children for several years, adults…
Read MoreA Bold Step Forward: Breakthrough in Gene Therapy for NF1
By Karissa Haberkamp | November 18, 2025 Researchers supported by NTAP have announced an exciting breakthrough in gene therapy for NF1. For the first time, scientists have created a smaller, working version of the NF1 gene, called a “mini-NF1.” They have packaged it into a specially engineered viral vector that can target tumor cells more…
Read MoreThe NF1 Cognitive Aging Project
Participants 40+ May Earn $40. Florida State University College of Medicine is conducting a study to investigate cognitive patterns and predictors within middle-aged and older adults with NF1.
Read MoreSMART-NF1 Project
Parent’s May Earn $10. Kids 13-17 May Earn $210. Florida State University College of Medicine is conducting a study to improve understanding of cognitive, behavioral, and socioeconomic function in the daily lives of adolescents with NF1.
Read MoreNF1 PN Pain Scale Research Study
Offered by: National Institutes of Health (NCI)Conducted by: Dr. Pamela Wolters Recruiting for children (8-17) with NF1 and plexiform neurofibromas (pNFs) for a research study to complete pain questionnaires electronically over a two-week period from home. Pediatric participants must also have a parent/caregiver willing to complete questionnaires as part of the study. Please email the study coordinator,…
Read More