What’s happening in NF1 research today

NTAP’s Innovative Funding Approach Delivers Results

June 1, 2023

NTAP’s innovative funding approach was featured in an article,”Pedal to the Metal” on Inside Hopkins, the Johns Hopkins Medicine daily publication highlighting exceptional and impactful programs. “How an innovative funding approach is advancing research and […]

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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 […]

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2021 Francis S. Collins Scholars Program in Neurofibromatosis Clinical and Translational Research Award Recipients Named

July 28, 2021

The Neurofibromatosis Therapeutic Acceleration Program (NTAP) at the Johns Hopkins University School of Medicine is proud to announce that Dr. Nicole Brossier, Dr. Suganth Suppiah, and Dr. Harish Vasudevan have been selected as the 2021 […]

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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 […]

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US Breakthrough Therapy Designation in neurofibromatosis type 1 granted by the FDA for the MEK1/2 inhibitor Selumetinib

April 23, 2020

On April 1, 2019, AstraZeneca (the maker of selumetinib) announced that the US Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) for the MEK 1/2 inhibitor selumetinib for the treatment of paediatric patients aged three […]

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Selumetinib in Children with Inoperable Plexiform Neurofibromas

April 22, 2020

N Engl J Med 2020; 382:1430-1442 DOI: 10.1056/NEJMoa1912735 List of authors. Andrea M. Gross, M.D., Pamela L. Wolters, Ph.D., Eva Dombi, M.D., Andrea Baldwin, P.N.P., Patricia Whitcomb, R.N., Michael J. Fisher, M.D., Brian Weiss, M.D., […]

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Selumetinib: The First Effective Treatment for Plexiform Neurofibromas

April 22, 2020

A young patient with NF1 who was treated at NCI gathers with her mother and members of NCI’s Pediatric Oncology Branch. Credit: National Institutes of Health Over 30 years of NCI-led and NCI-supported research culminated […]

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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. […]

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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 […]

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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 […]

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Induced pluripotent stem cells have been generated for the first time from tumor cells in order to study therapies for tumors developed in patients with hereditary diseases with predisposition to cancer

February 13, 2019

Induced pluripotent stem cells have been generated for the first time from tumor cells in order to study therapies for tumors developed in patients with hereditary diseases with predisposition to cancer  The study focuses on […]

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Induced Pluripotent Stem (iPS) Cells Generated from Human NF1 Associated Plexiform Neurofibromas made available to the research community

January 7, 2019

Neurofibromas are tumors of the peripheral nerves that are found most commonly in people with neurofibromatosis type I (NF1), a neurogenetic disease with an estimated birth incidence of 1/2500-3000.  A type of neurofibroma called a […]

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