Plexiform neurofibromas research

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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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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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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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 […]

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