“Being Okay” – Kylie’s NF1 Journey
When we are faced with an obstacle, big or small, we supposedly pick one of two options: to fight it or to run away. Nowadays, they say there are more…
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“Being Okay” – Kylie’s NF1 Journey
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NDA for NF1-PN Treatment Charges Ahead with Priority Review
According to a press release from GlobeNewswire, the biopharmaceutical company SpringWorks Therapeutics recently announced that the US Food and Drug Administration (FDA) has granted the company's New Drug Application (NDA)…
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NDA for NF1-PN Treatment Charges Ahead with Priority Review
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Neurofibromatosis Type 1: A Mother’s Struggle Living with Thousands of Tumors
According to a story from StokeonTrentLive, 34-year-old mother Rachel Potter is living with thousands of tumors thanks to a rare genetic disorder called neurofibromatosis type 1 (NF1). Though they aren't…
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Neurofibromatosis Type 1: A Mother’s Struggle Living with Thousands of Tumors
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“An Event to End NF” Helped Teen Raise Funds, Awareness for Neurofibromatosis
On May 17, 2024, Ohio was graced with a unique, Wizard of Oz-themed fundraising event called "An Event to End NF." NF, in this case, stands for neurofibromatosis, a rare genetic…
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“An Event to End NF” Helped Teen Raise Funds, Awareness for Neurofibromatosis
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)
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.…
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Rare Community Profiles: A Mother-Son Pair Discuss the Importance of Wellbeing in Managing NF2-Related Schwannomatosis (NF2)
Mom Advocates for More Research After Daughter’s NF1 Diagnosis
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…
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Mom Advocates for More Research After Daughter’s NF1 Diagnosis
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TRIAL RESULTS: How Did Mirdametinib Perform for NF1-PN?
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…
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TRIAL RESULTS: How Did Mirdametinib Perform for NF1-PN?
A Mother Raises NF1 Awareness After Son’s Diagnosis
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…
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A Mother Raises NF1 Awareness After Son’s Diagnosis
This Mom Ran the NY Marathon to Raise Neurofibromatosis Awareness
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…
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This Mom Ran the NY Marathon to Raise Neurofibromatosis Awareness
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In 10 Years, “Links for Lauren” Has Raised $500K for Neurofibromatosis
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…
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In 10 Years, “Links for Lauren” Has Raised $500K for Neurofibromatosis
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Rare Classroom: 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…
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Rare Classroom: Neurofibromatosis Type 2
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Feb 11: Cupid’s Undie Run Raises Neurofibromatosis Funds, Awareness
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…
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Feb 11: Cupid’s Undie Run Raises Neurofibromatosis Funds, Awareness
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May is Neurofibromatosis Awareness Month: Spreading Rare Disease Awareness
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…
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May is Neurofibromatosis Awareness Month: Spreading Rare Disease Awareness
Study for Vision Loss from NF1 Optic Pathway Glioma Funded By Gilbert Family Foundation
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).…
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Study for Vision Loss from NF1 Optic Pathway Glioma Funded By Gilbert Family Foundation
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Mom Runs NY Marathon to Fundraise for 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…
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Mom Runs NY Marathon to Fundraise for Neurofibromatosis
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NFX-179 Granted Orphan Drug Status for Cutaneous NF1
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…
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NFX-179 Granted Orphan Drug Status for Cutaneous NF1
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Koselugo Now EU Approved for NF1 and PN
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…
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Koselugo Now EU Approved for NF1 and PN
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This Mom is Helping Organize a Fundraiser for Neurofibromatosis in South Dakota
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…
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This Mom is Helping Organize a Fundraiser for Neurofibromatosis in South Dakota
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How Rare Disease Research Has Been Affected by the Pandemic
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…
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How Rare Disease Research Has Been Affected by the Pandemic
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FDA Approves a Treatment for Neurofibromatosis Type 1 for the First Time Ever
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…
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FDA Approves a Treatment for Neurofibromatosis Type 1 for the First Time Ever
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Planting Love Around the World: A Young Patient with Neurofibromatosis Works to Reduce COVID-19 Anxiety
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…
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Planting Love Around the World: A Young Patient with Neurofibromatosis Works to Reduce COVID-19 Anxiety
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NF1 Patients Still Need Proper Treatments
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…
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NF1 Patients Still Need Proper Treatments
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Even in Death, This Glioblastoma Patient’s Selfless Lego Donation Drive Touches Thousands of Lives
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…
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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
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,…
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17 Year Old With Grapefruit Sized Tumor Shares Her Story With Neurofibromatosis
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This Mom Raising a Son with Neurofibromatosis Started a Foundation to Support Parents
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…
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This Mom Raising a Son with Neurofibromatosis Started a Foundation to Support Parents
