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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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).…
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…
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…
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…
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…
Neurofibromatosis Neurofibromatosis is a rare condition that presents in different forms. Neurofibromatosis type one (NF1) causes tumors to grow on the plexiform neurofibromas, a type of nerve in the body. These tumors…
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…
According to a recent announcement by AstraZeneca, the U.S. FDA granted its approval for Koselugo (selumetinib). This is the first drug to be approved for the treatment of children…
According to BioSpace, healthcare technology company RDMD has raised $14 million in Series A financing, and developed a partnership with UCB. In addition to growing their platform to discuss…
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…
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…
According to a story from Esmo, the results of a recent phase 2 clinical trial bode well for pediatric and adolescent patients with neurofibromatosis type 1 (NF-1), a rare genetic…
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…
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,…
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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