Bringing Brigatinib to Neurofibromatosis Type-2 Patients: From...
Children’s Tumor Foundation and Takeda Pharmaceuticals Launch INTUITT for NF2 in Partnership with 6 Leading Medical Centers(PRWeb May 29, 2020)Read the full story at https://www.prweb.com/releases/bringing_brigatinib_to_neurofibromatosis_type_2_patients_from_promising_potential_to_platform_clinical_trial/prweb17157522.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - May 29, 2020 Category: Pharmaceuticals Source Type: news

New targets for childhood brain tumors identified
(Washington University School of Medicine) People with the genetic condition neurofibromatosis type 1 (NF1) are prone to developing tumors on nervous system tissue. A new study from Washington University School of Medicine in St. Louis has found that the development and growth of such tumors are driven by nearby noncancerous neurons and immune cells. The findings point to potential new therapeutic targets for people with NF1. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - May 1, 2020 Category: International Medicine & Public Health Source Type: news

FDA Approves Koselugo for Pediatric Neurofibromatosis Type 1
Title: FDA Approves Koselugo for Pediatric Neurofibromatosis Type 1Category: Health NewsCreated: 4/14/2020 12:00:00 AMLast Editorial Review: 4/15/2020 12:00:00 AM (Source: MedicineNet Cancer General)
Source: MedicineNet Cancer General - April 15, 2020 Category: Cancer & Oncology Source Type: news

FDA Approves Koselugo for Pediatric Neurofibromatosis Type 1
TUESDAY, April 14, 2020 -- Koselugo (selumetinib) has received the first approval for treatment of neurofibromatosis type 1 (NF1) in children ages 2 years and older, the U.S. Food and Drug Administration announced Friday. Approved specifically for... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - April 14, 2020 Category: General Medicine Source Type: news

FDA Approves Koselugo (selumetinib) for Pediatric Patients with Neurofibromatosis Type 1 Plexiform Neurofibromas
13 April 2020 -- AstraZeneca and MSD Inc., Kenilworth, N.J., US (MSD: known as Merck& Co., Inc. inside the US and Canada) today announced that the US Food and Drug Administration (FDA) has approved the kinase inhibitor Koselugo (selumetinib)... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - April 14, 2020 Category: Drugs & Pharmacology Source Type: news

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

Man with rare 'Elephant Man' disease gets six pounds of fleshy tumors surgically removed
Enrique Galvan, 27, from Asuncion, Paraguay, was born with a rare, genetic disease known as neurofibromatosis, which causes large, benign tumors to grow on nerve tissue. (Source: the Mail online | Health)
Source: the Mail online | Health - June 11, 2019 Category: Consumer Health News Source Type: news

Nerve fibers in skin involved in initiating neurofibromas in patients with disfiguring NF1
(Children's Tumor Foundation) A study published today in PLOS ONE discovered the origin of severely disfiguring masses of cells, called neurofibromas, that gradually develop throughout the skin of patients afflicted with Neurofibromatosis 1 (NF1). The discovery consisted of micro-lesions that contained small pathological clusters of nerve fibers and associated Schwann cells that proliferate to form the bulk of the disfiguring neurofibromas. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - May 21, 2019 Category: International Medicine & Public Health Source Type: news

What Causes Macrocephaly?
Discussion “Macrocephaly is defined as [an occipitofrontal circumference or head circumference, OFC ] of> 2 standard deviations above the mean or above the 97th percentage for a given age, and gender, or when serial measurement shows progressive enlargement, crossing of one or more major percentiles, or when there is an increase in OFC> 2 cm/month in the first 6 months of life.” Megalencephaly is enlargement of the brain parenchyma. The OFC should be measured using a non-elastic tape which surrounds the head along the line of the glabella and posterior occipital protrusion. There are ethnic differences f...
Source: PediatricEducation.org - May 20, 2019 Category: Pediatrics Authors: pediatriceducationmin Tags: Uncategorized Source Type: news

The husband and wife with THOUSANDS of tumours between them
Gail Appelgre, 58, and her other half Tim Golumbia, 55, of Edmonton in Alberta, Canada, have neurofibromatosis, which causes painful masses to grow along their nerve tissue. (Source: the Mail online | Health)
Source: the Mail online | Health - May 10, 2019 Category: Consumer Health News Source Type: news

AAP Updates Guidance for Care in Neurofibromatosis Type 1
WEDNESDAY, April 24, 2019 -- Guidance has been updated for the health supervision of children with neurofibromatosis type 1 (NF1), according to a clinical report from the American Academy of Pediatrics published online April 22 in Pediatrics. David... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - April 24, 2019 Category: Pharmaceuticals Source Type: news

Proximity biotinylation identifies a set of conformation-specific interactions between Merlin and cell junction proteins
Neurofibromatosis type 2 is an inherited, neoplastic disease associated with schwannomas, meningiomas, and ependymomas and that is caused by inactivation of the tumor suppressor gene NF2. The NF2 gene product, Merlin, has no intrinsic catalytic activity; its tumor suppressor function is mediated through the proteins with which it interacts. We used proximity biotinylation followed by mass spectrometry and direct binding assays to identify proteins that associated with wild-type and various mutant forms of Merlin in immortalized Schwann cells. We defined a set of 52 proteins in close proximity to wild-type Merlin. Most of t...
Source: Signal Transduction Knowledge Environment - April 23, 2019 Category: Science Authors: Hennigan, R. F., Fletcher, J. S., Guard, S., Ratner, N. Tags: STKE Research Resources Source Type: news

Adam Pearson neurofibromatosis: What is the genetic disorder? Signs and symptoms revealed
ADAM PEARSON is appearing on BBC ’s Pointless this afternoon, as the British actor joins hosts Alexander Armstrong and Richard Osman. Pearson has the genetic disorder neurofibromatosis, but what is it, and what are the signs and symptoms? (Source: Daily Express - Health)
Source: Daily Express - Health - April 21, 2019 Category: Consumer Health News Source Type: news

Selumetinib granted US Breakthrough Therapy Designation in neurofibromatosis type 1
AstraZeneca and MSD, Inc., Kenilworth, NJ, US (MSD: known as Merck & Co., Inc. inside the US and Canada) today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for the MEK 1/2 inhibitor and potential new medicine selumetinib. (Source: World Pharma News)
Source: World Pharma News - April 2, 2019 Category: Pharmaceuticals Tags: Featured AstraZeneca Business and Industry Source Type: news

Woman whose life is being ‘consumed’ by her giant leg a
Karina Rodini, from Sao Paolo, Brazil, has type one neurofibromatosis. Doctors haven't managed to get the condition under control, and her tumours now weigh 40kg (88.8lbs). (Source: the Mail online | Health)
Source: the Mail online | Health - March 29, 2019 Category: Consumer Health News Source Type: news

Woman, 40, has 'heavy' tumour engulfing her face that is inoperable
Aline Cerqueria Leite, from Brazil, has undergone 14 surgeries for neurofibromatosis, an incurable condition. But now, the risks are too high as the tumour has spread to her eyes, jaw and nose. (Source: the Mail online | Health)
Source: the Mail online | Health - March 14, 2019 Category: Consumer Health News Source Type: news

Woman, 28, has huge uncontrollable tumours on her legs and hips
Karina Rodini, from Sao Paolo, Brazil, has type one neurofibromatosis, a condition which makes non-cancerous tumours grow along someone's nerves and cannot be cured. (Source: the Mail online | Health)
Source: the Mail online | Health - January 31, 2019 Category: Consumer Health News Source Type: news

Mice sleeping fitfully provide clues to insomnia
(Washington University School of Medicine) Researchers at Washington University School of Medicine in St. Louis -- working with mice with sleep problems similar to those experienced by people with the genetic disease neurofibromatosis type 1 (NF1) -- believe the animals will help shed light on insomnia linked to NF1 or other factors. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 9, 2019 Category: International Medicine & Public Health Source Type: news

$12 million coming to UAB for research into genetic disorder
Four researchers at the UAB School of Medicine will put a deep focus on a genetic disorder over the next three years. The Gilbert Family Foundation will allocate $12 million over the next three years to UAB researchers exploring developmental and possibly curative therapies to address the underlying genetic abnormalities of neurofibromatosis type 1 (NF1), a multisystem genetic disorder. The incoming funding is part of the Gilbert Family Foundation’s Gene Therapy Initiative. The goal of the… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 1, 2019 Category: Biotechnology Authors: Tyler Patchen Source Type: news

$12 million coming to UAB for research into genetic disorder
Four researchers at the UAB School of Medicine will put a deep focus on a genetic disorder over the next three years. The Gilbert Family Foundation will allocate $12 million over the next three years to UAB researchers exploring developmental and possibly curative therapies to address the underlying genetic abnormalities of neurofibromatosis type 1 (NF1), a multisystem genetic disorder. The incoming funding is part of the Gilbert Family Foundation’s Gene Therapy Initiative. The goal of the… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 1, 2019 Category: Pharmaceuticals Authors: Tyler Patchen Source Type: news

Synodos does it again: Breaking barriers to solve the 'impossible' problems
(Children's Tumor Foundation) Treatment for low-grade gliomas in patients living with neurofibromatosis type 1 are now one step closer thanks to recent research discoveries initiated and funded by the Children's Tumor Foundation. The Foundation's SYNODOS consortium has just recently been published in Nature Medicine, showing that immunotherapy has the potential to impact gliomas. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - December 27, 2018 Category: Cancer & Oncology Source Type: news

What Causes Hyperpigmentation?
Discussion Skin color is primarily due to genetic factors. Melanocytes are usually found in skin in the basal layer of the epidermis which is also where the melanin usually lies. There are a range of normal skin phenotypes that have been described by Fitzpatrick and range from I-VI: I – pale white skin, blond hair, blue eyed, does not tan, always burns II – fair skin, blue eyed, tans poorly, burns easily III – darker white skin, burns initially then tans IV – light brown skin, tans easily, burns minimally V – brown skin, tans darkly easily, burns rarely VI – dark brown skin, alway...
Source: PediatricEducation.org - December 17, 2018 Category: Pediatrics Authors: pediatriceducationmin Tags: Uncategorized Source Type: news

Open science and data initiative announced for neurofibromatosis
(Children's Tumor Foundation) The Children's Tumor Foundation, together with the Neurofibromatosis Therapeutic Acceleration Program and Sage Bionetworks, has announced the first-ever open data portal for scientific research results in the field of neurofibromatosis (NF). The NF Data Portal marks the major first milestone in all three organizations' commitment to the development of the larger NF Open Science Initiative (NF-OSI), which draws experts from across research disciplines and disease areas in order to develop treatments for NF. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 14, 2018 Category: International Medicine & Public Health Source Type: news

Some brain tumors may respond to immunotherapy, new study suggests
(Columbia University Irving Medical Center) A new study suggests that a slow-growing brain tumor arising in patients affected by neurofibromatosis type 1 (NF1) may be vulnerable to immunotherapy, which gives the immune system a boost in fighting cancer. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 10, 2018 Category: International Medicine & Public Health Source Type: news

Mother who is covered in tumours has accepted her looks after her son was born with the disorder
Vanessa Cornelio, 25, from Glendale, Arizona, was born with neurofibromatosis, which causes tumours to grow on nerve tissue. Her two-year-old son Daniel Jr. inherited the condition. (Source: the Mail online | Health)
Source: the Mail online | Health - November 6, 2018 Category: Consumer Health News Source Type: news

Pakistani man covered in hundreds of bubble-like tumours
Ameer Ali, from Sehwan Sharif in Pakistan's Sindh province, has had small tumours on his abdomen and back since his childhood. Doctors believe he has a severe case of neurofibromatosis. (Source: the Mail online | Health)
Source: the Mail online | Health - October 8, 2018 Category: Consumer Health News Source Type: news

Pakistani man, 32, covered in hundreds of bubble-like tumours
Ameer Ali, from Sehwan Sharif in Pakistan's Sindh province, has had small tumours on his abdomen and back since his childhood. Doctors believe he has a severe case of neurofibromatosis. (Source: the Mail online | Health)
Source: the Mail online | Health - October 8, 2018 Category: Consumer Health News Source Type: news

Social worker who is covered from head to toe in tiny tumours has been refused into swimming pools
Gail Appelgren, 57, from Edmonton in Alberta, Canada, has battled with neurofibromatosis her whole life, which she believes makes her 'special' despite cruel comments from strangers. (Source: the Mail online | Health)
Source: the Mail online | Health - September 6, 2018 Category: Consumer Health News Source Type: news

Social worker who is covered from head to toe in tiny tumours has been refused into swimming pools
Gail Applegren, 57, from Edmonton in Alberta, Canada, has battled with neurofibromatosis her whole life, which she believes makes her 'special' despite cruel comments from strangers. (Source: the Mail online | Health)
Source: the Mail online | Health - September 5, 2018 Category: Consumer Health News Source Type: news

Screening for Neuroendocrine Tumors Is Recommended for NF1 Patients
A study on pheochromocytoma and paraganglioma associated with neurofibromatosis type 1 highlights how screening for these malignancies is important. (Source: CancerNetwork)
Source: CancerNetwork - August 28, 2018 Category: Cancer & Oncology Authors: Dave Levitan Source Type: news

Selumetinib granted orphan designation in Europe for neurofibromatosis type 1
AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US (known as MSD outside the US and Canada) announced that the European Medicines Agency (EMA) has granted orphan designation to selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1). (Source: World Pharma News)
Source: World Pharma News - August 9, 2018 Category: Pharmaceuticals Tags: Featured AstraZeneca Business and Industry Source Type: news

Indian teen is unable to walk due to her 'defunct' claw-like fist
Mita Sabar, 18, from a remote village in Odisha, east India, is thought to be suffering from plexiform neurofibromatosis, which means her hand is so heavy she is unable to lift it. (Source: the Mail online | Health)
Source: the Mail online | Health - August 8, 2018 Category: Consumer Health News Source Type: news

The teenager with a 22lb hand: 18-year-old is unable to walk due to her 'defunct', claw-like fist  
Mita Sabar, 18, from a remote village in Odisha, east India, is thought to be suffering from plexiform neurofibromatosis, which means her hand is so heavy she is unable to lift it. (Source: the Mail online | Health)
Source: the Mail online | Health - August 8, 2018 Category: Consumer Health News Source Type: news

Children's Tumor Foundation Drug Discovery Awards announced
(Children's Tumor Foundation) The Children's Tumor Foundation (CTF), the largest non-governmental funder of neurofibromatosis (NF) research, and PLOS ONE, a leading peer-reviewed scientific journal, are pleased to announce the successful completion of the first funding cycle of the Drug Discovery Initiative Registered Report (DDIRR) 2017 Awards, a funder-publisher partnership integrating the Registered Reports model into the grant application process. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 11, 2018 Category: International Medicine & Public Health Source Type: news

Indian grandfather's tumours left him unable to eat, talk or speak
Subhash Chand, 54, from Uttar Pradesh state in India, suffers from a condition known as Neurofibromatosis or Von Recklinghausen's Disease. He developed the disorder at just 20 years old. (Source: the Mail online | Health)
Source: the Mail online | Health - May 15, 2018 Category: Consumer Health News Source Type: news