Seeking participants for NF1 study of how cutaneous neurofibromas form

The Neurofibromatosis Therapeutic Acceleration Program (NTAP) at Johns Hopkins University seeks participants study that will use a specialized 3D photography system to take whole-body pictures once a year for up to five years to track the progression of cutaneous neurofibromas (cNF) over time.

Neurofibromatosis type 1 (NF1) diagnosis fuels nurse educator’s passion for goal concordant care

Tori McClosky’s senior year of high school turned upside down when a tumor was found on her brain. The cause was NF1. In the 20 years since then, McClosky has become a nurse and patient at MD Anderson and her experience has fueled her passion for patient care.

Breakthrough in Neurofibromatosis Type 1 Treatment: SpringWorks Therapeutics' Mirdametinib Shows Promise

Explore the groundbreaking Phase 2b ReNeu trial by SpringWorks Therapeutics, showcasing the potential of mirdametinib in transforming the treatment landscape for Neurofibromatosis Type 1.

University of Minnesota's Patient Perspective Study for Adults with NF1

Researchers at the University of Minnesota are conducting qualitative study to better understand experiences of families in terms of accessing or receiving treatments for behavioral health concerns. Learn more and see if you qualify.

Stanford University's NF1 Optic Glioma Study

Researchers at Stanford University's Byers Eye Institute Ophthalmic Innovation Program are seeking participants for an NF1 vision study. Learn more and see if you qualify to help researchers identify biomarkers for visual restoration strategies in NF1-type optic pathway gliomas.

Identification of Pre-Malignant Lesions In Pediatric Patients With NF1

A pilot study is using MRI scans of pediatric patients to screen for markers that predict cancerous tumors as well as benign tumors capable of malignant transformation. When combined with machine-learning algorithms informed by both retrospective and prospective scans, investigators believe the technique could revolutionize proactive management and preventive interventions for cancer predisposition syndromes.

Opportunity to Participate in an Independent Research Study to Tell Your NF1-PN Story

Bryter, an independent research company, is conducting a research study to understand about the lives of those (caregivers / patients) living with NF1-PN. The research is being sponsored by a pharmaceutical company and is designed to give the NF1-PN community the opportunity to tell their story about the lived experience.

Alexion's Ongoing Research on Plexiform Neurofibromas (NF1-PNs)

Alexion is conducting two ongoing studies focusing on adults with NF1 with plexiform neurofibromas (NF1-PNs). Each study seeks to understand a different element regarding plexiform neurofibromas and potential treatment options. The first focuses on pediatric patients taking selumetinib, and the second focuses on challenges in treatment and pain management.

NF1 Research Study: Perceived Stress & Coping in Adults with NF1

Are you an adult with NF1? Researchers at Vanderbilt are asking the question: “does a reported family history of NF1 influence the levels of perceived stress and/or coping styles of adults with NF1?” Learn more about the study and how to participate.

The NF Network Releases Volume 22 of The Network Edge

In partnership with NF Northeast, the NF Network is delighted to bring you Volume 22 of our science column, The Network Edge, written by science writer Vanessa L. Merker, PhD. This periodic research review presents a summary of recent highlights from NF research and clinical trials in bite-sized pieces, allowing you to focus on the topics of most interest to you.

FY23 CDMRP Funding Overview: Neurofibromatosis Research Program

Each year in February, the NF Network Advocacy Program gathers on Capitol Hill in Washington, D.C. to advocate for continued federal funding for NF research through the Congressionally Directed Medical Research Program (CDMRP-NFRP). Read more on this fiscal year's funding focus.

The NF Network Releases Volume 21 of The Network Edge

In partnership with NF Northeast, the NF Network is delighted to bring you Volume 21 of our science column, The Network Edge, written by science writer Vanessa L. Merker, PhD. This periodic research review presents a summary of recent highlights from NF research and clinical trials in bite-sized pieces, allowing you to focus on the topics of most interest to you.

NF1 Cutaneous Neurofibroma Consortium Project

PolyGenRX, in partnership with The University of Newcastle and several NF1 researchers, is conducting a new NF1 project. Adults with NF1 are invited to participate in the NF1 cutaneous neurofibroma (cNF) consortium project. Findings will aim to understand the root cause of these tumors and potential treatment options for cNFs.

NFlection Therapeutics Seeking Adult Patients for Investigational Study on Cutaneous Neurofibromas (cNFs)

NFlection Therapeutics, Inc., a clinical-stage biopharmaceutical company, is currently recruiting for NFX-179 for the treatment of cutaneous neurofibromas. NFX-179 is a topical, first-in-class, “soft” MEK inhibitor that is currently being evaluated in Phase 2 clinical trials in patients with NF1.

Recursion Announces Initiation of Phase 2/3 Trial for the Treatment of NF2-Mutated Meningiomas

Recursion has announced the initiation of its Phase 2/3 POPLAR-NF2 clinical trial evaluating REC-2282: a potentially first-in-disease treatment for progressive neurofibromatosis type 2 (NF2)-mutated meningiomas.

Epilepsy drug stops nervous system tumor growth in mice

In a recent study, researchers at Washington University School of Medicine in St. Louis have discovered that neurons carrying a mutation in the NF1 gene are hyperexcitable and suppressing this hyperactivity with lamotrigine stops tumor growth in mice. Hear from senior author, Dr. David Gutmann.

Stanford Conducting Remote Study for Adults with NF1

Stanford University is seeking participants for a free, remote genetic testing study. This research will study the genes of adults with neurofibromatosis type (NF1) to help researchers understand why some are more likely to develop certain disorders and manifestations, such as increased cutaneous neurofibromas.

Targeting the Immune System Blocks Optic Glioma Formation in Mice

Researchers in the Gutmann Laboratory at Washington University used mice genetically engineered to develop optic gliomas - a type of tumor that can affect children with NF1. Postdoctoral fellows, Drs. Jit Chatterjee and Amanda Costa, discovered an exciting finding involving immune cell function and how it affects tumor growth.

Raise NF Awareness on Rare Disease Day

Rare Disease Day is February 28th! It is estimated that there are over 300 million people living with rare diseases around the globe. Among those 300 million, over 130,000 Americans alone are living with neurofibromatosis. Join us in raising NF awareness and generating change in the rare disease community.

A new resource for managing hypertension in children with NF1

A collaborative of specialists from around the globe has come together with a vision to help patients with pediatric Renovascular Hypertension (pRVH) get the best care possible. This diverse and comprehensive team of researchers, clinicians, patients and families will improve the quality of life and treatment outcomes for pRVH patients.

NF2 Natural History Study at the National Institutes of Health

The National Institutes of Health (NIH) is the taxpayer funded federal agency responsible for medical research. The benefit of their research is returned to taxpayers. NIH has a long history of conducting research in NF1 and NF2. John and Linda Manth enrolled their daughter Leah 15 years ago and share information on a revised prospective Natural History Study for NF2.

NFlection Therapeutics Seeking Adult Participants for Clinical Trial on Cutaneous Neurofibromas (cNFs)

NFlection Therapeutics, Inc., a clinical-stage biopharmaceutical company, is currently recruiting for NFX-179 for the treatment of cutaneous neurofibromas. NFX-179 is a topical, first-in-class, “soft” MEK inhibitor that is currently being evaluated in Phase 2 clinical trials in patients with NF1.

In a common genetic disorder, blood test reveals when benign tumors turn cancerous

Individuals with NF1 often develop non-cancerous tumors that grow along nerves. Researchers from the NCI Center for Cancer Research and Washington University School of Medicine in St. Louis have developed a blood test that, they believe, could one day offer a highly sensitive and inexpensive approach to detect cancer early in people with NF1. The blood test could also help doctors monitor how well patients are responding to treatment for their cancer.

Mouse Study Reveals Rare Genetic Disorder May Be Improved by Hypertension Drug

NF2 is a hereditary condition most commonly associated with bilateral vestibular schwannomas (VS), also known as acoustic neuromas. Although these tumors are benign, they can cause hearing and balance problems. Now, a mouse study by scientists at Massachusetts General Hospital (MGH) and Massachusetts Eye and Ear reveals that the blood pressure drug, losartan, may benefit patients with NF2.

Pain in NF1: iCanCope Mobile Application Study

Dr. Frank Buono's DoD-funded study sets out to understand the impact of the iCanCope - NF mobile application on individuals suffering from chronic pain due to NF1, with the goal of reducing pain symptoms. If you are currently experiencing NF1 pain, you are invited to participate in this clinical trial.

Optic nerve firing may spark growth of vision-threatening childhood tumor

Researchers have shown in a recent study of mice how the act of seeing light may trigger the formation of optic gliomas in young children who are born with NF1. The research team, funded by the NIH, discovered that the neural activity which underlies these signals can both ignite and feed the tumors.

Stem Cells May Explain Why Rare Inherited Tumor in Kids Is So Aggressive

Researchers from Memorial Sloan Kettering reported they have discovered that a subpopulation of cells within MPNSTs that could explain why the tumors may come back after treatment. The research, which was carried out in mice, suggests that scientists eventually could be able to create targeted therapies for the treatment of people with MPNSTs.

Brigitte Widemann Named a Top Ten Clinical Research Achievement Awardee

Brigitte C. Widemann, M.D., Chief of the Pediatric Oncology Branch at the National Cancer Institute, has been named a Top Ten Clinical Research Achievement Awardee by the Clinical Research Forum and has received a Distinguished Clinical Research Achievement Award for her study “Selumetinib in Children with Inoperable Plexiform Neurofibromas.”

Cabozantinib for NF1-Related Plexiform Neurofibromas: A Phase 2 Trial

Based on preclinical observations, the Neurofibromatosis Clinical Trials Consortium (NFCTC) launched a clinical trial to determine the response rate of cabozantinib on NF1-PNs in patients 16 years and older. The phase 2 trial showed promising success, saying lower doses of cabozantinib may be optimal for the NF1 population and still lead to therapeutic response.

2020 CDMRP NF Research Highlight: Pain in Neurofibromatosis

For the past several years, the Neurofibromatosis Research Program (NFRP) has listed pain as an area of emphasis in its program funding opportunities, encouraging research applications aimed at addressing this critical need in the NF community. Each year, pain research continues to progress with new studies supported by the NFRP.

MGH Seeking Participants for Resiliency for NF Studies

Researchers at the Integrated Brain Health Clinical and Research Program at Massachusetts General Hospital are seeking participants for their studies, comparing 2 stress management programs for adults and adolescents with NF to see which one best improves quality of life and well-being for those living with NF.

The MEK inhibitor selumetinib may reduce spinal neurofibroma burden in patients with NF1 and plexiform neurofibromas

In a new publication, researchers detail the positive effect the MEK inhibitor, selumetinib, has on reducing the spinal neurofibroma burden in patients with NF1 and plexiform neurofibromas.

Technion discovers how to reduce cognitive damages of brain diseases

Technion-Israel Institute of Technology, in partnership with Cincinnati Children’s Hospital Medical Center (CCHMC), found out how to rescue functional and structural connectivity, and reduce cognitive impairments of brain diseases with drug treatment.

Please take this new NF survey for adults!

You are being invited to participate in an anonymous online survey to help researchers design psychosocial trials for adults with neurofibromatosis. We also have some important questions on how the current coronavirus pandemic is affecting you.

New Targets for Childhood Brain Tumors Identified

Findings at the Washington University School of Medicine in St. Louis indicates neurons and immune cells work together to promote tumor growth in neurofibromatosis type 1 (NF1). These findings point to new potential treatments for low-grade brain tumors in people with NF1.

FDA Approves First Treatment for Neurofibromatosis

The U.S. Food and Drug Administration has approved Koselugo (selumetinib) to treat plexiform neurofibromas in pediatric patients with NF1 ages 2 years and older. This approval means Koselugo is the first approved treatment for neurofibromatosis!

FDA Approves First Therapy for Children with Debilitating and Disfiguring Rare Disease

The U.S. Food and Drug Administration has approved Koselugo (selumetinib) to treat plexiform neurofibromas in pediatric patients with NF1. This approval means Koselugo is the first approved treatment for neurofibromatosis.

New Research Study for Understanding Chronic Pain and Work Barriers within NF Population

Dr. Frank Buono leads a new research study investigating the impact of NF pain and, as a result, the quality of life and work-related barriers.

Encouraging News for Kids with Neurofibromatosis Type 1

30 years ago, Dr. Collins led a team that discovered the gene that underlies NF1. Today, he is pleased to share what promises to be the first effective treatment to help young people affected by NF1.

In NIH trial, selumetinib shrinks tumors, provides clinical benefit for children with NF1

In a trial sponsored by the NCI Cancer Therapy Evaluation Program (CTEP) and conducted by the NIH intramural program, the drug selumetinib improved outcomes for children with NF1 and plexiform neurofibromas.

Neurofibromatosis gene provides new insight into breast cancer resistance to hormone therapy

An international team of researchers led by scientists at Baylor College of Medicine has new insights into the function of neurofibromin, a tumor suppressor produced by the NF1 gene.

Bevacizumab Treatment Reportedly Increases Quality of Life in Adults & Children

Study Finds That Bevacizumab Treatment for NF2-Related Vestibular Schwannomas Increases Reported Quality of Life in Adults and Children.

SpringWorks' NF1 Plexiform Neurofibroma Trial

The ReNeu trial is a Phase 2b clinical trial designed to evaluate the safety, effectiveness, and tolerability of an investigational medicine called mirdametinib in children 2 years or older and adults with an inoperable NF1-PN.

DoD-Funded Study to Evaluate Pain Reduction in Adults with NF1

You are invited to participate in a group based focus group to discuss your chronic pain due to Neurofibromatosis Type 1 (NF1).

New Research Study at UMN and UWM for Children and Adolescents with NF1

Researchers at University of Minnesota, Twin Cities and University of Wisconsin, Madison are combining efforts to better understand the strengths and difficulties of children and adolescents with NF1.

The NFRP Presents the Fiscal Year 2018 Early Investigator Research Award Recipients

The Neurofibromatosis Research Program (NFRP) Early Investigator Research Award (EIRA) mechanism was offered for the first time in fiscal year 2018 (FY18).

SpringWorks Therapeutics' ReNeu Trial Press Release

SpringWorks Therapeutics Announces Initiation of Phase 2b ReNeu Clinical Trial of Mirdametinib in Children and Adults with Neurofibromatosis Type 1 (NF1)-Associated Plexiform Neurofibromas (NF1-PN)

New marker for tumor aggression in NF1

New molecular insights could mean more accurate prognoses and better selection of therapies.

Long-Term Outcomes of Stereotactic Radiosurgery...

The study confirmed a lack of significant difference in outcomes of SRS between NF2-associated and sporadic VSs.

A Greater Understanding of Tumor Cells

Through better understanding of the mechanical processes at work in tumors, Kristen Mills, an assistant professor of mechanical, aerospace, and nuclear engineering at Rensselaer Polytechnic Institute, hopes to support the development of more effective treatments.

SpringWorks Therapeutics Gains FDA Endorsement for Tumor Drug

Biotech firm SpringWorks Therapeutics has gained an endorsement from the U.S. Food and Drug Administration to help expedite the review of a drug that would treat rare tumors.

Couple give embryos, 'hope' to UM in fight against rare disease

The Cellucci family has donated seven embryos with the NF2 gene to the University of Michigan so researchers could create an embryonic stem cell line that other researchers could study, with the hope of one day discovering a breakthrough.

Investigating the Role of Fibroblasts in the Pathogenesis in Neurofibromas

With support from an Exploration-Hypothesis Development Award through the NFRP, Dr. Lu Le and his team are investigating the role of fibroblasts in the tumor microenvironment and hippo signaling pathway in neurofibromas.

Westerville father spearheads effort to fund research for daughter's disorder

The work of NF2 BioSolutions is now paying off in a big way.

Mueller Lab for Gene Therapy

Being moved by the impact of NF2, Dr Mueller decided to run a gene therapy pilot to identify if his lab’s gene therapy platform could be a good candidate for tackling the NF2 mutation.

New study reveals treatment pathway for neurofibromatosis

James Wymer, M.D. and his team have identified a novel pathway for potential treatment of NF.

Cerebellopontine Angle (CPA) Model

A Novel Tool for Investigating Immunotherapy in Neurofibromatosis Type 2 Vestibular Schwannomas

Dr. Vincent Riccardi Video

Dr. Vincent Riccardi is gaining momentum through Innovation and recruitment.

The Merlin Interactome Identifies Cell Junction Proteins

Proximity biotinylation identifies a set of conformation-specific interactions between Merlin and cell junction proteins

TOP NEWS: US FDA Grants Breakthrough Status To AstraZeneca Treatment

The drug selumetinib has been granted a breakthrough therapy designation in the US for neurofibromatosis type 1.

Neurofibromatosis Research Program Announcements

The NF Network plays a vital role in securing federal funds for Neurofibromatosis Research. One of the federal programs funded is the Congressionally Directed Medical Research Program, specifically the Neurofibromatosis Research Program Check out the NF Research Program Announcements for 2019.

Mice Sleeping Fitfully Provide Clues To Insomnia

Mice that sleep fitfully could help researchers unravel the mystery of insomnia.

Marker found for condition that causes tumors

UT Southwestern researchers have made a major advance in uncovering the biology.

25th Annual NF Center Research Symposium

Washington University marked 25 years of NF research by hosting the NF Center Research Symposium.

Clinical response to MEK inhibitor I in a NF1 patient

Ecancer medical science reported their August 2018 issue about a case of a 12 year old child with NF1. 

NF Center Researchers Identify Key Role for Immune Cells in Brain Tumors

In a new study they now show that microglia can be programmed to support tumor formation and growth by T cells.

Brain tumors occur often in kids with common genetic syndrome

Neurologists have estimated that only 15 to 20 percent of kids with NF1 develop brain tumors.

Fiscal Year 2016 New Investigator Award Recipients

The Neurofibromatosis Research Program (NFRP) utilizes the New Investigator Award (NIA) to introduce the next generation of investigators and their ideas to the NF research community.

Confronting the Challenge of Neurofibromatosis

Neurofibromatosis as a name encompasses at least three diseases in fact. They are called neurofibromatosis type one, neurofibromatosis type two, and Schwannomatosis.

Genomic Roadmap for Sarcoma Established

This landmark study, involving Drs. Brian Van Tine, David H. Gutmann and Li Ding from Washington University, characterized over 200 adult sarcomas representing six different types of these cancers.

Surgeons Preserve Patients’ Hearing with Innovative Brainstem Implant

FDA-approved device helps patients with neurofibromatosis type 2

New guidelines for the clinical care of adults with NF1

American College of Medical Genetics and Genomics convened experts to write new guidelines

Halting Intracranial Tumor Growth

Promising treatment identified for tumor that causes hearing loss and sometimes death.

Study finds key risk factor for severe NF1 symptoms

Research led by Ludwine Messiaen, professor of genetics at the University of Alabama at Birmingham, found a key risk factor for severe symptoms of NF1.

Turning piglets into personalized avatars for sick kids

A team of scientists wants to accelerate research into a genetic disorder by using CRISPR to copy unique mutations from affected children into pigs.

The Specific Genetic Mutation May Influence NF1 Brain Tumor Development

Researchers at the Washington University NF Center found that the location of the NF1 gene mutation correlates with the development of brain tumors.

Study finds increased breast cancer risk

A new study published by Dovepress suggests women with neurofibromatosis type 1 begin screening for breast cancer at age 30 due to increased risk between ages 30 and 50.

New cell of origin for optic gliomas identified

A new study revealed information about the development of optic gliomas, a type of tumor children with NF1 are susceptible to.

Genetic tests + coordinated care + research = HOPE

Life with an inherited disease sometimes brings unexpected twists and turns. Five-year-old Gus Erickson has navigated the gyrations with the help of Mayo Clinic’s Neurofibromatosis Clinic.

New research offers hope to neuro-tumor patients

New research could offer hope to the thousands of people affected by Neurofibromatosis 2.

Participate in NF research

Did you know that there are 167 clinical trials for neurofibromatosis (NF) in various stages of completion? People with NF can play a critical role in the development of new treatments and the search for a cure by participating in clinical trials.

Washington U professor receives $3.5M grant

David H. Gutmann, director of the Neurofibromatosis Center at the Washing University School of Medicine in St. Louis, was awarded a $3.5 million grant to study NF1.

Drug shrinks pediatric neural tumors

In an early-phase clinical trial, in most cases, children with neurofibromatosis type 1 and plexiform neurofibromas responded with tumor shrinkage.

Swedish teen walks using 3D printed hip implant

Fanny Fellesen, a Swedish 16-year-old with neurofibromatosis, was told she would never walk again.

Drug shows promise with hearing loss

A drug already in use for some cancers is not only halting hearing loss in some patients, but reversing it.

New drug approved for NF2 treatment

BioXcel announced the U.S. Food and Drug Administration has granted Orphan Drug Designation to BXCL101 for the treatment of Neurofibromatosis Type 2.

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