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What’s happening in NF1 research today
Koselugo (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 […]
Read MoreData made available for Research Tools that Accelerate Development of Therapies for Neurofibromatosis Type 1 Plexiform Neurofibromas
For Release: October 2018 Development of therapies for human disease remains dependent on well-characterized and validated model systems. Slow growing histologically benign tumors such as neurofibromas are challenging to grow in typical culture conditions. As […]
Read MoreData Available to Accelerate Development of Therapies for Neurofibromatosis Type 1 Plexiform Neurofibromas
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 […]
Read MoreData made available for a 3D model of Neurofibromatosis Type 1 Plexiform Neurofibromas
For Release: October 2018 Development of therapies for human disease remains dependent on well-characterized and validated model systems for pre-clinical therapeutic testing. Most in vitro drug testing is performed on cells growing in two dimensions […]
Read MoreTwo New Clinician-Scientists Chosen for Neurofibromatosis Award, Research Support
Author: Cathy Gara. July 2018–The Neurofibromatosis Therapeutic Acceleration Program (NTAP) at the Johns Hopkins University School of Medicine is proud to announce that Dr. Shruti Garg, of the University of Manchester, and Dr. Ina Ly, […]
Read MoreFunding Available for Neurofibromatosis Research from the Department of Defense – Congressionally Directed Medical Research Programs
The FY18 Neurofibromatosis Research Program (NFRP) is accepting applications for research projects. Applications to the Fiscal Year 2018 (FY18) Neurofibromatosis Research Program (NFRP) are being solicited for the Defense Health Agency (DHA) J9, Research and Development […]
Read MoreCutaneous Neurofibromas Initiative Outlines Current Biology and Path Forward for Clinicians and Scientists
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. […]
Read MoreNew Research Tools Available in the Search for Therapies for Neurofibromatosis Type 1 Plexiform Neurofibromas
New Research Tools Available in the Search for Therapies for Neurofibromatosis Type 1 Plexiform Neurofibromas By Cathy Gara | For Release: June 15, 2018 Plexiform neurofibromas affect up to 50 percent of people with neurofibromatosis […]
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