A team of investigators from the NTAP gene therapy replacement initiative published their groundbreaking gene replacement strategy for Neurofibromatosis type 1 (NF1) — one of the most common single-gene disorders worldwide. Lead author, Dr. Renyuan Bai, and team […]
Every person living with neurofibromatosis type 1 (NF1) has a story—and nearly all of those stories include cutaneous neurofibromas (cNFs). These benign tumors of the skin may not pose a life-threatening risk, but for the […]
It can spawn tumors throughout the nervous system and bone deformities that riddle the whole body with pain. One of the most common rare diseases in the world, the genetic condition neurofibromatosis type 1, or NF1, can cause a wide spectrum of mild to severe symptoms—all stemming from a single mutation on a solitary gene: the NF1 gene on chromosome 17.
In response to a critical funding gap affecting the NF1 research community, the Neurofibromatosis Therapeutic Acceleration Program (NTAP) has launched a new training initiative: the NF1 Basic and Translational Laboratory Scholars Program. This program is […]
The first manuscript from the NTAP 2022 Cutaneous Neurofibroma Symposium was published digitally by the Journal of Investigative Dermatology today. The paper RAS Signaling Gone Awry in the Skin: The Complex Role of RAS in […]
For Immediate Release: April 10, 2020 Today, the U.S. Food and Drug Administration approved Koselugo (selumetinib) for the treatment of pediatric patients, 2 years of age and older, with neurofibromatosis type 1 (NF1), a genetic […]
News Release Regulatory News Service 13 April 2020 07:00 BST First medicine approved to treat this rare and debilitating genetic condition AstraZeneca and MSD Inc., Kenilworth, N.J., US (MSD: known as Merck & Co., Inc. […]
NCI Press Release, Posted: March 18, 2020, Contact: NCI Press Office 240-760-6600 Dr. Brigitte Widemann with Travis Carpenter, who received selumetinib for NF1 at NIH. Credit: National Cancer Institute Findings from a phase 2 clinical trial show […]
Patient derived tissues are a critical tool to support the ongoing research that improves our understanding of disease formation mechanisms, and helps drive the discovery of new therapeutics. As part of its commitment to providing […]
For Release: October 2018 Neurofibromas are tumors of the nerve that are found most commonly in people with neurofibromatosis type I (NF1). NF1 is a neurogenetic disease with an estimated prevalence of 1/2500-1/3000. A type […]
