FDA Approves Koselugo for Pediatric Neurofibromatosis Treatment FDA Approves Koselugo for Pediatric Neurofibromatosis Treatment
The approval of selumetinib was based on results from the phase 2 SPRINT Stratum trial investigating the efficacy of the drug on kids who have NF1 with symptomatic, inoperable plexiform neurofibromas.FDA Approvals (Source: Medscape Hematology-Oncology Headlines)
Source: Medscape Hematology-Oncology Headlines - April 13, 2020 Category: Cancer & Oncology Tags: Neurology & Neurosurgery News Source Type: news
FDA approves selumetinib for neurofibromatosis type 1 with symptomatic, inoperable plexiform neurofibromas
Oncology / Hematology News Burst (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - April 13, 2020 Category: Drugs & Pharmacology Authors: FDA Source Type: news
FDA approves first ever treatment for neurofibromatosis
(Children's Tumor Foundation) The FDA has approved Koselugo (selumetinib) for use in patients with inoperable plexiform neurofibromas, a common manifestation in the disease neurofibromatosis type one (NF1). The FDA's approval of AstraZeneca's and MSD (Merck)'s submission is a major milestone for patients living with neurofibromatosis (NF), a genetic disorder that causes tumors to grow on nerves throughout the body. Affecting 1 in 3,000 people of all populations equally, this announcement is the first ever approved treatment for NF. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - April 13, 2020 Category: Cancer & Oncology Source Type: news
New Drug Selumetinib Helps Shrink Inoperable Tumors in Kids
FRIDAY, March 20, 2020 -- A new trial confirms that the drug selumetinib shrinks tumors in children suffering from neurofibromatosis type 1.
The condition is characterized by changes in skin coloring and the growth of tumors along nerves in the... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - March 20, 2020 Category: General Medicine Source Type: news
In NIH trial, selumetinib shrinks tumors, provides clinical benefit for children with NF1
(NIH/National Cancer Institute) Findings from a phase 2 clinical trial show that the drug selumetinib improves outcomes for children with the genetic disorder neurofibromatosis type 1 (NF1). In the trial, selumetinib shrank the inoperable tumors that develop with NF1 called plexiform neurofibromas, and children experienced reduced pain, improved function, and better overall quality of life after receiving the treatment. The trial was led by intramural researchers at the National Cancer Institute, part of the National Institutes of Health. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - March 18, 2020 Category: Cancer & Oncology Source Type: news
Neurofibromatosis gene provides new insight into breast cancer resistance to hormone therapy
(Baylor College of Medicine) Neurofibromin (NF1), a protein involved in cancer growth, helps estrogen receptor-positive (ER+) breast cancer develop resistance to hormone therapy by two independent mechanisms, not one as previously thought. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - March 5, 2020 Category: International Medicine & Public Health Source Type: news
'Bubble skin man' in India ravaged by HUNDREDS of tumours over his body
Diwakar Bisoye, 65, from Odisha State, is covered in benign tumours of varying sizes. He suffers with suspected neurofibromatosis, a genetic and incurable illness. (Source: the Mail online | Health)
Source: the Mail online | Health - January 6, 2020 Category: Consumer Health News Source Type: news
Mother, 35, covered head-to-toe in thousands of tumours
Ashley Jernigan, from Montgomery, Alabama, suffers from neurofibromatosis. The genetic disorder struck her at puberty and quickly left her body covered in thousands of lumps. (Source: the Mail online | Health)
Source: the Mail online | Health - January 2, 2020 Category: Consumer Health News Source Type: news
Children's Tumor Foundation and DELopen increase access to compounds for neurofibromatosis
(Children's Tumor Foundation) The Children's Tumor Foundation (CTF), an innovative and global neurofibromatosis research foundation announced today a collaboration with DELopen, a DNA-encoded library technology platform sponsored by WuXi AppTec, a leading global provider of R&D and manufacturing services enabling companies in the pharmaceutical, biotechnology and medical device industries. DELopen will provide researchers free access to billions of molecules to seek insights into and develop therapies for the treatment of NF under grants covered by CTF's Discovery Fund. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 19, 2019 Category: International Medicine & Public Health Source Type: news
US FDA Accepts Regulatory Submission of New Drug Application for Selumetinib in Neurofibromatosis Type 1 (NF1) and Grants Priority Review
KENILWORTH, N.J.--(BUSINESS WIRE)--AstraZeneca and Merck (NYSE:MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) and granted priority... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - November 14, 2019 Category: Drugs & Pharmacology Source Type: news
FDA accepts submission for selumetinib as treatment for neurofibromatosis
(Children's Tumor Foundation) The FDA granted acceptance to file status for selumetinib, for use in patients with plexiform neurofibromas, a common manifestation in neurofibromatosis type one (NF1). This is a key milestone for becoming the first approved treatment for NF. This follows previous designations granting orphan drug status as well as breakthrough status, and enters the drug into the FDA's approval pipeline. The FDA also granted priority review, and a PDUFA has been set for Q2 of 2020. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 14, 2019 Category: International Medicine & Public Health Source Type: news
Rate and frequency of bullying victimization in school-age children with neurofibromatosis type 1 (NF1) - Holland AA, Stavinoha PL, Swearer SM, Solesbee C, Patel S, Klesse LJ.
Children and adolescents with the genetic, tumor predisposition syndrome neurofibromatosis type I (NF1) have varying degrees of physical stigmata characteristic of the disease and experience high rates of social difficulties. The present study was the firs... (Source: SafetyLit)
Source: SafetyLit - November 11, 2019 Category: International Medicine & Public Health Tags: Age: Adolescents Source Type: news
Selumetinib Clinically Improves NF1-Associated Neurofibromas
The MEK inhibitor selumetinib showed clinical responses in the plexiform neurofibromas of adult patients with neurofibromatosis type 1. (Source: CancerNetwork)
Source: CancerNetwork - October 29, 2019 Category: Cancer & Oncology Authors: Seth Augenstein Source Type: news
Light brown skin speckles on Oklahoma girl, 4, turn out to be a warning sign of a brain tumor
Charlie Thomas, four, from Moore, Oklahoma, was diagnosed with neurofibromatosis, which causes large tumors to grow on nerve tissues, including on her optic nerve and brain (Source: the Mail online | Health)
Source: the Mail online | Health - September 25, 2019 Category: Consumer Health News Source Type: news
Woman, 20, with a 100° vertebrae was given just 24hours to live
Katie Krzyzanowski was born with neurofibromatosis, which causes tumours to form on nerve endings. This led the now 20-year-old, of Somerset, to develop scoliosis at four years old. (Source: the Mail online | Health)
Source: the Mail online | Health - June 17, 2019 Category: Consumer Health News Source Type: news
