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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