NF1 research Article Archives

Ras Signaling Gone Awry: New Research Published on the Role of Ras in Cutaneous Neurofibroma Pathogenesis

May 27, 2023

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 […]

FDA Approves First Therapy for Children with Debilitating and Disfiguring Rare Disease

April 27, 2020

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 […]

Koselugo (selumetinib) approved in US for paediatric patients with neurofibromatosis type 1 plexiform neurofibromas

April 13, 2020

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. […]

In NIH trial, selumetinib shrinks tumors, provides clinical benefit for children with NF1

March 18, 2020

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 […]

Driving NF1 Research: High Quality, Clinically Annotated Tissue and biospecimen materials Available from the Johns Hopkins NF1 Biospecimen Repository

February 25, 2019

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 […]

Data Available to Accelerate Development of Therapies for Neurofibromatosis Type 1 Plexiform Neurofibromas

October 22, 2018

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 […]

Cutaneous Neurofibromas Initiative Outlines Current Biology and Path Forward for Clinicians and Scientists

July 13, 2018

July 10, 2018: One of the hallmarks of the complex syndrome Neurofibromatosis type 1 (NF1) are cutaneous neurofibromas (cNF). cNF are tumors involving the skin that affect up to 99 percent of adults with NF1. […]

Cutaneous Neurofibroma Initiative Projects (2018)

April 1, 2018

9 Projects Transforming the Field of NF1 Research NTAP convenes symposiums on a regular basis to determine research priorities, define new avenues of inquiry and develop requests for applications (RFAs) in neurofibromatosis type-1 (NF1) research […]

NTAP 2018 Cutaneous Neurofibroma Initiative: 4 Supplement Paper summaries

February 20, 2018

As a result of the 2018 Cutaneous Neurofibroma Initiative, several working groups were formed to produce papers addressing key challenges identified. Those papers are summarized below with links to the full papers provided. This information […]

NTAP 2016 Cutaneous Neurofibroma Symposium

January 5, 2017

Establishing priorities and advancing the field of NF1 research Introduction to NTAP symposiaNTAP convenes leading researchers and innovators from across multiple fields and scientific backgrounds in regular symposia to establish priorities for the future NF1-related […]