In The News

Koselugo Fasting Update

Koselugo - the first FDA-approved treatment shown to reduce the size of plexiform neurofibromas (PN) - has made lifechanging impacts on NF1-patients and caregivers. New improvements could result in no longer needing to fast to accommodate treatment.

SketchNF: My Colorful Life with Neurofibromatosis

In an effort to spread joy and awareness on neurofibromatosis to children and young adults with NF, SketchNF has created a coloring book that illustrates several real stories. Each book purchased allows SketchNF to donate 2 books back to over 30 clinics across the country.

‘So proud’ - Matthew’s award triumph is a first for county Scouts

A SCOUT from Warwickshire has been awarded the prestigious Cornwell Scout Badge for his outstanding courage even in the face of significant health challenges.

Spanish Study: Finding Medical Information in NF1

Researchers are seeking Spanish-seeking patients and caregivers with NF1 to share their experience in accessing medical information. See if you qualify by completing a short survey.

Bringing new voices to the rare disease community

PatientWorthy is helping bring a voice to the rare disease community with their rare disease podcast, “Wait How Do You Spell That?” There, the podcasters talk about issues affecting people, rare and underdiagnosed conditions, and interview advocates from across the country.

Dr. Robert Kesterson works to overcome major hurdles in cancer research and rare diseases

Dr. Bob Kesterson is Professor of Cancer Precision Medicine and Director of the Genetically Engineered Models core facility at Pennington Biomedical. Families with children diagnosed with NF1 reach out to him in hopes of developing the best therapy for their child.

Dan Gilbert donates nearly $400M to fund neurofibromatosis facility in Detroit

In honor of their late son, Dan and Jennifer Gilbert made a significant contribution to create a specialized NF facility in Detroit as a part of Henry Ford Health's campus expansion.

Little girl donates two feet of her hair in incredible sponsored charity chop

Young Stella Dance knows firsthand how it feels to receive support from a special charity. Living with NF1, Stella wanted to give back to a special organization who helped her live out her dreams as a little girl. Her big salon appointment resulted in 2 less feet of hair and an incredible fundraising opportunity.

Man who wears prosthetic eye due to rare condition hopes his story can inspire others

Amit Ghose, 33, from Birmingham, was diagnosed with NF1 when he was 2 years old. The rare genetic condition caused tumors to grow on the left side of his face and eye, resulting in the removal of his eye at 11. Now, he's using his platform on TikTok to educate, share, and inspire people through his story.

SketchNF: Empowering the Pediatric NF Community

After Sahil Shah was diagnosed with NF1, he sought ways to connect within the NF community. As a young adolescent, he found a need for additional support for others his age. Wanting to get involved and raise funds and awareness for neurofibromatosis, the idea for SketchNF was born from a conversation with his brother and a unique fundraiser, Doodle4NF.

Dr. Gutmann Elected to National Academy of Medicine

Dr. David Gutmann, founder of the Washington University NF Center and NF Hope Concert Advocate of Hope Award Winner, was recently elected to the National Academy of Medicine. This election is one of the highest honors for contributions to health and medicine.

NF1 SLEEP STUDY: Understanding the Link between Neurofibromatosis and Sleep

Dr. Richa Saxena and her research team at Massachusetts General Hospital are conducting a study to learn more about sleep, circadian rhythm, quality of life, genetics, and other functions in patients with NF1. Learn if you qualify for this virtual study and how you can participate.

Vanderbilt Studying Reading Challenges in NF1

Researchers at Vanderbilt University are seeking to understand challenges our NF community faces in reading comprehension and new ways to teach reading-related skills. Children and adults who qualify for the study will be introduced to a reading tutoring program and 1 of 2 groups will also receive a medication, Lovastatin.

Miller School Selected for National NF2 Tumor Clinical Trial

A University of Miami Miller School of Medicine researcher is hoping an innovative multicenter clinical trial will lead to new therapies for NF2. Their goal is to accelerate the process of identifying effective drugs that will address the unique needs of this patient population.

Gilbert Family Foundation Invests $18M Toward Gene Therapy Initiative

The Gilbert Family Foundation, a private fund established by Dan and Jennifer Gilbert, announced Thursday it is funding more than $18 million in grants toward NF1 research. The money will fund a three-year campaign as part of the organization's Gene Therapy Initiative.

NF1 Cutaneous Neurofibroma Study: Optical Imaging of cNFs

Dr. Wangcun Jia is an Associate Researcher at the Beckman Laser Institute and Medical Clinic at University of California, Irvine. You are invited to participate in this DoD-funded clinical study which aims to detect early-stage cutaneous neurofibromas (cNF) using noninvasive optical imaging technologies - before they become visible on the skin surface

FREE CME Webinar: NF1 and Plexiform Neurofibromas with Dr. Bruce Korf

PeerView with the support from AstraZeneca is hosting a free Continuing Education webinar to inform medical professionals and physicians on Neurofibromatosis Type-1 and currently approved and emerging targeted therapies for NF1. The NF Network encourages our NF community to share this information with their physicians and medical staff.

Camp New Friends Returns This Summer

After experiencing the fun of summer camp virtually, Camp New Friends returns to bring “a fun, supportive environment for kids with neurofibromatosis.” Designed to reduce the social stigma and feelings of isolation associated with NF, campers will enjoy summer fun and social connections.

SpringWorks Therapeutics Announces Full Enrollment of Phase 2b ReNeu Trial Evaluating Mirdametinib in Patients with NF1-PNs

SpringWorks Therapeutics has completed enrollment for their phase 2b ReNeu trial evaluating mirdametinib, an investigational MEK inhibitor, in NF1 patients with plexiform neurofibromas.

The NF Network Releases Volume 20 of The Network Edge

In partnership with NF Northeast, the NF Network is delighted to bring you Volume 20 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.

Recursion is Granted FDA Fast Track Designation for REC-2282 for the Potential Treatment of NF2-Mutated Meningiomas

Recursion, a clinical-stage biotechnology company, announced that the FDA has granted the company Fast Track designation for the investigation of REC-2282. REC-2282 is an HDAC inhibitor being developed for the treatment of NF2-mutated meningiomas.

Gilbert Family Foundation Funds Study to Understand Vision Loss from NF1-Optic Pathway Glioma

The Gilbert Family Foundation, a private nonprofit established by Jennifer and Dan Gilbert to accelerate a cure for NF1, announced funding of a new $5.4M clinical study. The study, led by investigators at Children’s Hospital of Philadelphia and the Stanford University School of Medicine, aims to understand the mechanisms of vision loss caused by NF1-associated optic pathway gliomas.

SpringWorks Conducting Several Studies with MEK Inhibitor, Mirdametinib

SpringWorks Therapeutics is conducting a clinical trial to study an investigational MEK inhibitor called mirdametinib in people with a plexiform neurofibroma (PN) associated with NF1. The study, called the ReNeu trial, is currently enrolling people ages 2 to 17. The purpose is to determine the effectiveness of mirdametinib at improving a PN that is causing significant issues in people with NF1.

NFlection Therapeutics Announces Orphan Drug Designation of NFX-179 for the Treatment of Cutaneous Neurofibromas

NFlection Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing novel drug candidates for rare RASopathies, announced the FDA has granted orphan drug designation for NFX-179 - a topical, first-in-class, “soft” MEK inhibitor that is currently being evaluated in Phase 2 clinical trials in patients with cutaneous neurofibromas.

Revised diagnostic criteria for NF1: an international consensus recommendation

By incorporating major developments in genetics, ophthalmology, dermatology, and neuroimaging, new research has resulted in the revision of diagnostic criteria for neurofibromatosis type 1 (NF1).

FY22 House Appropriations Bill Includes $20M for NF Research

Thanks to your advocacy efforts, we are pleased to announce that the House Appropriations Committee has included $20M for the Neurofibromatosis Research Program (NFRP) in their Fiscal Year 2022 Defense spending bill! The NF Network extends our deepest gratitude to our letter signers, advocates, and supporters.

The NF Network Releases Volume 19 of The Network Edge

In partnership with NF Northeast, the Neurofibromatosis Network is delighted to bring you Volume 19 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 easily find and focus on the topics of most interest to you.

Thank You FY22 House “Dear Colleague” Letter Signers

Upon completing our Virtual Hill Visits back in February, we asked for your help in contacting members of the House of Representatives. Your requests asked for support of continued federal funding for NF research, and we are proud to announce this year’s FY22 House “Dear Colleague” letter signers.

Update on NF1 Research from SpringWorks Therapeutics

SpringWorks Therapeutics is currently conducting a clinical trial for an investigational MEK inhibitor called mirdametinib in patients with NF1-associated plexiform neurofibromas. Their update includes an FAQ with information on mirdametinib and key information on clinical trials.

IQVIA Conducting Study for Individuals with NF1 and Plexiform Tumors

IQVIA, a healthcare research firm, is currently conducting a new research project measuring pain associated with plexiform neurofibromas. By participating, you will help researchers understand the complexity and pain associated with PNs, which may lead to new treatments.

NF1 Survey: Communication, Swallowing, and Hearing for Children and Individuals with NF1

California State University seeks to learn how communication, hearing, and swallowing difficulties impact individuals with NF1.

NF1 Research Study: iCanCope Mobile Application

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.

The REINS Collaboration Recruiting Patient Representatives

As patients and caregivers of those living with neurofibromatosis, the REiNS Collaboration seeks your help in the fight to cure all types of NF by helping to design clinical trials. Joining the patient representative program is a chance for you to make a difference in finding treatments for NF.

The NF Network Releases Volume 18 of The Network Edge

The Neurofibromatosis Network is delighted to bring you Volume 18 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 easily find and focus on the topics of most interest to you.

University of Utah Conducting Study for Adults with NF1

Researchers at The University of Utah, Division of Medical Genetics are seeking participants for an ongoing study for adult patients with NF1. The trial will evaluate the effect of Vitamin D supplementation and bone mass in participants in the study.

Entertainers Share Talent and Support During NF Hope Concert: The LIVE Interviews

Members of the entertainment community sought out ways to help the NF cause following the NF Hope Concert Live Stream. With the overwhelming support, Jeff Leibow launched The LIVE Interviews.

Inspire Hosts “Summertime Life Hacks: Kids with Special Needs”

Inspire believes that “Together, We’re Better.” Providing online support communities for families and individuals around the world, Inspire seeks to bring a sense of hope and peace of mind through their summer discussion series.

CFC Opens Special Solicitation for COVID-19

In light of the ongoing pandemic, members of several federal departments and agencies sought ways to do more to help during COVID-19. The Combined Federal Campaign has opened a special solicitation to aid CFC-approved charities as they face unprecedented challenges.

From Graduate to Advocate

Rachel Sakofs, a Speech Language Pathologist and alum of MGH Institute of Health Professions, uses her IHP education to help her son and raise awareness of his rare disorder.

Local teachers speak out in Washington for a cure for a rare disease

Stephanie Ham and fellow first-grade teacher Lauren Hanrahan recently went to Washington, D.C. to fight for funding for a rare disease, Neurofybromatosis Type 2, which Hanrahan has.

The Nation’s Largest Clinic for Neurofibromatosis Helps a Brave Boy Avert Life with a Breathing Tube

Dani and John Boyer knew there was a 50–50 chance that their children might inherit John’s disease.

Two Local Teachers Raise Awareness to Spread Kindness and Make Change

A video has gone viral in support of NF awareness thanks to these two friends.

Perceived Fatigue in Children and Young Adults with NF1

Investigators sought to understand and explain the prevalence and severity of perceived fatigue in children and young adults affected by neurofibromatosis type 1.

Kim Bischoff, Executive Director of NF Network, is a #TCFGoalGetter

#TCFGoalGetters don’t just dream big, but take the leap to pursue their passion. Check out Kim’s story and her inspiration behind her work at the NF Network.

Regina Vasquez Runs 5K to Support Neurofibromatosis Research

South Florida Realtor Regina Vasquez, of Beachfront Realty, participates in the Neurofibromatosis Network #NFStrong 5K for Neurofibromatosis.

Pigs Help Kids Fight NF1

Cutting edge research being done at the UW-Madison NF research facility means hope for the Geier family.

You’re Perfect! - A Mother's Journey

‘I need to tell you something.’ He teared up. ‘How do we fix it?’ I took my sweet little girl to the car, and buckled her in. ‘There’s nothing wrong with you. You’re perfect!’

Immune cells determine how fast certain tumors grow

Study suggests new approaches to treating NF1 brain tumors

Identical Twins Who Look Nothing Alike

Adam and Neil Pearson are identical twins, but you’d never know it from looking at them. Listen to their story in Jonathan Braue’s deeply affecting short documentary, The Pearson Twins.

Mom Blazes Path to Cure for Son’s Rare Disease

Nicole Henwood was told her son, A.J., had an incurable disease. She has set out to change that.

Neurofibromatosis Northeast Advocates for Funding

Karen Peluso of NF Northeast, joined 82 other individuals from 23 states to advocate for neurofibromatosis research funding.

$5.4 million coming to UAB for research into genetic disorder

The Gilbert Family is backing heavy research into gene therapy.

Fashion Blogger Uses Clothes to Hide Tumors

Model Arooj Aftab, 22, used oversized clothes to hide tumors on her hips.

Terminally Ill Gamer’s Wish Comes True

Rockstar Games have granted a terminally ill gamer’s wish to play their upcoming title.

Unique tricycle helps boy feel normal

AJ Stasio received the surprise of the summer when a stranger purchased an adult tricycle for him as a means of mobility.

World Record Holder Seeking Cure For NF

World Record Endurance Marathoner Becca Pizzi will run the Falmouth Road Race on Sunday, August 19th as part of the Neurofibromatosis Northeast Team.

Join two-time World Marathon champion Becca Pizzi for youth running camp

Two-time World Marathon Champ Becca Pizzi and ultra marathoner Scot DeDeo for the first annual Kids Running Camp.

Selumetinib Continues to Show Promise in Children with NF1

An investigational drug called selumetinib can shrink tumors in children and young adults with a genetic syndrome NF1.

Gov. Charlie Baker declares May NF Awareness Month

On May 1 Massachusetts Governor Charlie Baker and Lieutenant Governor Karyn Polito signed a proclamation officially declaring May Neurofibromatosis Awareness Month.

Kids Day: Team medical approach helps teen keep smiling

When Barbara Langdon's son, Finnian, was 4 months old, she found a pea-sized lump on his back.

Family, videography key for teen battling rare disease

Alex Leisenring, a high school senior, dreams of one day becoming a videographer.

This one's for Joan

Jeremy Clements, who drives the No. 51​​​​​​​ in the NASCAR Nationwide Series, wanted to do something special to honor a fan who recently passed away.

Harry Potter star's child diagnosed with NF1

Tansy Smethurst, whose mum Rakie Ayola plays Hermione Granger in the Harry Potter play, was on holiday when she suddenly developed spots on her body and needed to be rushed to hospital.

Patriots tight end makes special visit

Make-A-Wish transformed 7-year-old Ryan Feeney's backyard into a New England Patriots-themed football field, complete with Patriots tight end Rob Gronkowski.

Tim Tebow meets fan with NF during game

During a St. Lucie Mets game against the Charlotte Stone Crabs late last month, Tim Tebow made a young fan’s day.

Make-A-Wish throws party for NF teen

15-year-old Ayrianna Day Chief was treated a Hollywood party complete with performers and paparazzi courtesy of the Make-A-Wish Foundation.

I lost my voice, but help others find theirs

Being blind wasn't going to stop Alex Hubbard from becoming a journalist, but his NF2 diagnosis almost did.

Broadway stars raise funds for NF

Jeff Leibow has called upon Broadway friends including Chad Kimball and Mary Callanan to appear May 8 at a concert at New York’s SubCulture nightclub to raise money for the Neurofibromatosis Network.

Jeff Leibow shares personal mission

Jeff Leibow recently appeared on Good Morning Washington to raise awareness for neurofibromatosis.

Live life to the fullest

The trophies and scars Leah Manth has collected during her lifetime aren’t just rewards for participating in sports.

NF found in 15th century

A Renaissance painting in Mantua, Italy contains the portrait of a maidservant with neurofibromatosis.

Learning to love yourself

As a teenager, her mother told her, “Only someone kind would want to be with you.” Then, in her early 20s, Janet Miller began to notice small bumps on her skin.

Why this boy chose not to have his facial tumors removed

Ethan D’Amato was born with a number of facial tumors, but he chose to not have them removed rather than risk losing his ability to smile and laugh.

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