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What’s happening in NF1 research today
NTAP’s Innovative Funding Approach Delivers Results
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 […]
Read MoreRas Signaling Gone Awry: New Research Published on the Role of Ras in Cutaneous Neurofibroma Pathogenesis
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 […]
Read More2021 Francis S. Collins Scholars Program in Neurofibromatosis Clinical and Translational Research Award Recipients Named
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
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 […]
Read MoreUS Breakthrough Therapy Designation in neurofibromatosis type 1 granted by the FDA for the MEK1/2 inhibitor Selumetinib
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 […]
Read MoreSelumetinib in Children with Inoperable Plexiform Neurofibromas
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., […]
Read MoreSelumetinib: The First Effective Treatment for Plexiform Neurofibromas
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 […]
Read MoreKoselugo (selumetinib) approved in US for paediatric patients with neurofibromatosis type 1 plexiform neurofibromas
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. […]
Read MoreIn NIH trial, selumetinib shrinks tumors, provides clinical benefit for children with NF1
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 […]
Read MoreDriving NF1 Research: High Quality, Clinically Annotated Tissue and biospecimen materials Available from the Johns Hopkins NF1 Biospecimen Repository
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 […]
Read MoreInduced 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
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 […]
Read MoreInduced Pluripotent Stem (iPS) Cells Generated from Human NF1 Associated Plexiform Neurofibromas made available to the research community
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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