neurofibromatosis Archives

Jo and Oscar. Photo courtesy of Jo Ward

Rare Community Profiles: A Mother-Son Pair Discuss the Importance of Wellbeing in Managing NF2-Related Schwannomatosis (NF2)

Post author:Jessica Lynn
Post published:April 4, 2024
Post category:Neurofibromatosis Type 2

Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

Mom Advocates for More Research After Daughter’s NF1 Diagnosis

Post author:Jessica Lynn
Post published:December 21, 2023
Post category:ADHD Neurofibromatosis Neurofibromatosis Type 1

Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…

source: shutterstock.com

TRIAL RESULTS: How Did Mirdametinib Perform for NF1-PN?

Post author:Jessica Lynn
Post published:December 18, 2023
Post category:Neurofibroma Neurofibromatosis Neurofibromatosis Type 1

Over their lifetime, people with neurofibromatosis type 1 (NF1), a rare genetic disorder, have a 30-50% chance of developing plexiform neurofibromas. These tumors grow on peripheral nerve sheaths throughout…

A Mother Raises NF1 Awareness After Son’s Diagnosis

Post author:Jessica Lynn
Post published:November 28, 2023
Post category:Neurofibromatosis Neurofibromatosis Type 1

Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…

Photo by sporlab on Unsplash

This Mom Ran the NY Marathon to Raise Neurofibromatosis Awareness

Post author:Jessica Lynn
Post published:November 15, 2023
Post category:Neurofibromatosis

26.2 miles. 5 boroughs. And a deep feeling of fulfillment when she finally crossed the finish line. When Holly Griffin first decided to run the New York Marathon, she was…

Source: Pixabay.com

In 10 Years, “Links for Lauren” Has Raised $500K for Neurofibromatosis

Post author:Jessica Lynn
Post published:October 23, 2023
Post category:Neurofibromatosis Neurofibromatosis Type 1

Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…

source: shutterstock

Rare Classroom: Neurofibromatosis Type 2

Post author:James Moore
Post published:May 19, 2023
Post category:Neurofibromatosis Neurofibromatosis Type 2

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

Source: www.pixabay.com

Feb 11: Cupid’s Undie Run Raises Neurofibromatosis Funds, Awareness

Post author:Jessica Lynn
Post published:February 2, 2023
Post category:Neurofibromatosis

If you’ve ever wanted to run around Philadelphia in your underwear for a good cause, I have an event to tell you about! As first reported by the Philly…

source: pixabay.com

May is Neurofibromatosis Awareness Month: Spreading Rare Disease Awareness

Post author:James Moore
Post published:May 26, 2022
Post category:Neurofibromatosis Neurofibromatosis Type 1 Neurofibromatosis Type 2

According to a story from the Children's Tumor Foundation (CTF), the month of May is recognized as Neurofibromatosis (NF) Awareness Month. This is a time for spreading awareness about neurofibromatosis…

Study for Vision Loss from NF1 Optic Pathway Glioma Funded By Gilbert Family Foundation

Post author:Alyssa Stevens
Post published:December 1, 2021
Post category:Neurofibromatosis Neurofibromatosis Type 1 Neurofibromatosis Type 2

According to a recent article, the nonprofit Gilbert Family Foundation has offered to fund the $5.4 million clinical study to better understand vision loss from NF1-associated optic pathway gliomas (NF1-OPGs).…

skeeze / Pixabay

Mom Runs NY Marathon to Fundraise for Neurofibromatosis

Post author:Jessica Lynn
Post published:November 12, 2021
Post category:Neurofibromatosis

I've certainly never been a runner -- and neither was Diane Owens, at least for a while. The mother, who lives in Southbury, CT, admits that it used to be…

https://unsplash.com/photos/Rg0KmrUPB_Q

NFX-179 Granted Orphan Drug Status for Cutaneous NF1

Post author:Jessica Lynn
Post published:August 26, 2021
Post category:Neurofibromatosis Neurofibromatosis Type 1

According to a recent press release from biopharmaceutical company NFlection Therapeutics, Inc. (“NFlection”), the FDA granted Orphan Drug designation to the company’s drug candidate NFX-179. Altogether, this treatment is designed…

https://unsplash.com/photos/_jbClosDsD4

Koselugo Now EU Approved for NF1 and PN

Post author:Jessica Lynn
Post published:June 28, 2021
Post category:Neurofibromatosis Neurofibromatosis Type 1

On June 22, 2021, PharmaTimes Online reported that the European Commission (EC) granted conditional approval to Koselugo (selumetinib), an orally administered therapy, for use in pediatric patients with neurofibromatosis…

source: pixabay.com

This Mom is Helping Organize a Fundraiser for Neurofibromatosis in South Dakota

Post author:James Moore
Post published:July 13, 2020
Post category:Neurofibromatosis Rare Disease

According to a story from keloland.com, Westin Cuka, age six, was first diagnosed with neurofibromatosis, a rare disorder, three years ago. Since then, the family has traveled on multiple occasions…

source: pixabay.com

How Rare Disease Research Has Been Affected by the Pandemic

Post author:Trudy Horsting
Post published:May 15, 2020
Post category:Duchenne Muscular Dystrophy Neurofibromatosis

Maxwell and Riley Freed are twins who just celebrated their third birthday. Maxwell has a rare disease called SLC6A1. Sadly, this was a notable birthday because at 3, Maxwell will…

source: pixabay.com

FDA Approves a Treatment for Neurofibromatosis Type 1 for the First Time Ever

Post author:James Moore
Post published:April 15, 2020
Post category:Neurofibroma Neurofibromatosis Neurofibromatosis Type 1 Rare Disease

A press release from the US Food and Drug Administration (FDA) recently announced the approval of the drug selumetinib (marketed as Koselugo) for the treatment of patients aged two years…

source: pixabay.com

Planting Love Around the World: A Young Patient with Neurofibromatosis Works to Reduce COVID-19 Anxiety

Post author:Jessica Lynn
Post published:April 13, 2020
Post category:COVID-19 Neurofibromatosis Rare Disease

According to a story from Wexford People, 8-year-old Mia-Lily Ruttle wants to spread some hope to people around the globe. The girl, who has neurofibromatosis, was upset by the impact…

source: pixabay.com

NF1 Patients Still Need Proper Treatments

Post author:Patient Worthy Contributor
Post published:February 26, 2020
Post category:Neurofibromatosis Type 1 Rare Disease

By Danielle Bradshaw from In The Cloud Copy Neurofibromatosis type 1 (NF1)-related plexiform neurofibromas (pNFs) is a condition that many children struggle to manage due to a lack of proper…

source: pixabay.com

Even in Death, This Glioblastoma Patient’s Selfless Lego Donation Drive Touches Thousands of Lives

Post author:James Moore
Post published:December 16, 2019
Post category:Glioblastoma Neurofibromatosis

According to a story from 10tv.com, Kellan Shatto is an eight year old boy who really likes to play with Legos. He also has a rare disease: neurofibromatosis. The disease…

17 Year Old With Grapefruit Sized Tumor Shares Her Story With Neurofibromatosis

Post author:Sunniva Bean
Post published:December 4, 2019
Post category:Neurofibromatosis Rare Disease

As originally reported in Mirror, when Sian Hopper was in secondary school, she began to get headaches. She attributed them to stress: she had been studying for her GCSE exams,…

source: pixabay.com

This Mom Raising a Son with Neurofibromatosis Started a Foundation to Support Parents

Post author:James Moore
Post published:November 21, 2019
Post category:Neurofibromatosis

According to a story from Soweto Urban, mother Lebohang Leuta is no stranger to the challenges that come with raising and taking care of a child with a chronic rare…

RoyBuri / Pixabay

Scientists Are Using Pigs to Develop a More Useful Animal Model of Neurofibromatosis Type 1

Post author:James Moore
Post published:September 16, 2019
Post category:Neurofibromatosis

According to a story from wearegreenbay.com, a team of scientists associated with the University of Wisconsin, Madison are undertaking a project that will help improve the effectiveness of research related…

pixel2013 / Pixabay

Experimental Treatment for Neurofibromatosis Type 1 Earns Orphan Drug Designation in the EU

Post author:James Moore
Post published:August 2, 2019
Post category:Neurofibromatosis Neurofibromatosis Type 1

According to a story from BioPortfolio, the biopharmaceutical company SpringWorks Therapeutics, Inc., has recently announced that the European Commission has given the company's investigational drug candidate mirdametinib Orphan Drug designation.…

FotografieLink / Pixabay

Family Support: Twin Sister Carries Twins for Neurofibromatosis Type 2 Patient

Post author:James Moore
Post published:June 28, 2019
Post category:Neurofibromatosis Neurofibromatosis Type 2

According to a story from The Columbian, twins Jill Noe and Whitney Bliesner had developed a deep bond during their childhood days growing up in Oregon. However, they did not…

sasint / Pixabay

For This Man With Severe Neurofibromatosis, Surgery Feels Like a Second Chance

Post author:James Moore
Post published:June 20, 2019
Post category:Neurofibromatosis

According to a story from The Washington Post, Enrique Galvan has been subjected to relentless bullying and rejection ever since he first started school. The reason why? He was diagnosed…

Mom Advocates for More Research After Daughter’s NF1 Diagnosis

TRIAL RESULTS: How Did Mirdametinib Perform for NF1-PN?

A Mother Raises NF1 Awareness After Son’s Diagnosis

This Mom Ran the NY Marathon to Raise Neurofibromatosis Awareness

In 10 Years, “Links for Lauren” Has Raised $500K for Neurofibromatosis

Rare Classroom: Neurofibromatosis Type 2

Feb 11: Cupid’s Undie Run Raises Neurofibromatosis Funds, Awareness

May is Neurofibromatosis Awareness Month: Spreading Rare Disease Awareness

Study for Vision Loss from NF1 Optic Pathway Glioma Funded By Gilbert Family Foundation

Mom Runs NY Marathon to Fundraise for Neurofibromatosis

NFX-179 Granted Orphan Drug Status for Cutaneous NF1

Koselugo Now EU Approved for NF1 and PN

This Mom is Helping Organize a Fundraiser for Neurofibromatosis in South Dakota

How Rare Disease Research Has Been Affected by the Pandemic

FDA Approves a Treatment for Neurofibromatosis Type 1 for the First Time Ever

Planting Love Around the World: A Young Patient with Neurofibromatosis Works to Reduce COVID-19 Anxiety

NF1 Patients Still Need Proper Treatments

Even in Death, This Glioblastoma Patient’s Selfless Lego Donation Drive Touches Thousands of Lives

17 Year Old With Grapefruit Sized Tumor Shares Her Story With Neurofibromatosis

This Mom Raising a Son with Neurofibromatosis Started a Foundation to Support Parents

Scientists Are Using Pigs to Develop a More Useful Animal Model of Neurofibromatosis Type 1

Experimental Treatment for Neurofibromatosis Type 1 Earns Orphan Drug Designation in the EU

Family Support: Twin Sister Carries Twins for Neurofibromatosis Type 2 Patient

For This Man With Severe Neurofibromatosis, Surgery Feels Like a Second Chance

The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

“Shades of Psoriasis”: Plaque Psoriasis and Sharing Your Story

Understanding RSV: Why Combatting Ethnic and Age-Related Health Disparities Could Improve Outcomes

We Need Equity and Diversity Within Rare Disease Research

DNA in Color: How Ronya Nelson is Working to Close the Diversity Gap in Genetics - Part 1

Bridging the Diversity Gap in Healthcare

Congratulations to the 2023 Recipients of the Diversity in Lupus Research Awards!

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