“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
Parenting With Confidence: Practical Tips for Families Navigating Neurofibromatosis Type 1
Learning that your child has neurofibromatosis type 1 (NF1) can be overwhelming. NF1 affects each child differently and brings uncertainty, but with the right knowledge, resources, and support, parents can…
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Parenting With Confidence: Practical Tips for Families Navigating Neurofibromatosis Type 1
Gomekli: A Breakthrough in Treating Neurofibromatosis Type 1 with Plexiform Neurofibromas
Gomekli (mirdametinib) has become the first FDA-approved treatment for both adults and children with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas (PN)—tumors that often cannot be surgically removed. While…
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Gomekli: A Breakthrough in Treating Neurofibromatosis Type 1 with Plexiform Neurofibromas
Understanding Genetic Testing and Counseling for Neurofibromatosis Type 1
Neurofibromatosis Type 1 (NF1) is a genetic disorder diagnosed primarily through a set of clinical criteria established by the National Institutes of Health (NIH). Typically, a clinical diagnosis of NF1…
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Understanding Genetic Testing and Counseling for Neurofibromatosis Type 1
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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
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
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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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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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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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Study of the Week: New Genetic Predisposition Variants in Rhabdomyosarcoma
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
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Study of the Week: New Genetic Predisposition Variants in Rhabdomyosarcoma
A Med Student Assignment Became a Valuable Lesson
The winning entry for the 2019 Student Voice was published recently in the Orphanet Journal of Rare Diseases. The prize-winning article, a warm and personal account, was written by Annie…
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A Med Student Assignment Became a Valuable Lesson
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ICYMI: First Drug to be Approved for Neurofibromatosis Type 1 and Plexiform Neurofibromas (NF1-PN)
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…
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ICYMI: First Drug to be Approved for Neurofibromatosis Type 1 and Plexiform Neurofibromas (NF1-PN)
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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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MEK Inhibitor Successfully Treats Young Patients’ Neurofibromatosis Tumors
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…
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MEK Inhibitor Successfully Treats Young Patients’ Neurofibromatosis Tumors
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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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Experimental Treatment for Neurofibromatosis Type 1 Earns Orphan Drug Designation in the EU
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.…
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Experimental Treatment for Neurofibromatosis Type 1 Earns Orphan Drug Designation in the EU
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Children’s National Health System Researchers Receive Grants to Develop High-Tech Diagnostic Aids
According to a press release from the Children's National Health System (a children's hospital in Washington, D.C.), two scientists from the Institution have been selected to receive federal funding grants…
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Children’s National Health System Researchers Receive Grants to Develop High-Tech Diagnostic Aids
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Experimental Neurofibromatosis Type 1 Therapy Earns Fast Track Designation
According to a story from Global Genes, the drug developer SpringWorks Therapeutics announced that the US Food and Drug Administration (FDA) has granted the company Fast Track Designation for its…
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Experimental Neurofibromatosis Type 1 Therapy Earns Fast Track Designation
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Potential Treatment for Neurofibromatosis Type 1 Earns Fast Track Designation
According to a story from Acrofan, the biopharmaceutical company SpringWorks Therapeutics, Inc. has recently announced the that company's experimental product candidate PD-0325901 has earned Fast Track designation from the US…
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Potential Treatment for Neurofibromatosis Type 1 Earns Fast Track Designation
A Team of Scientists Researching A Cure for Neurofibromatosis Type 1
A three-year research grant of $11 million has been awarded through the Gilbert Family Foundation in America. According to a recent article in News-Medical Life Sciences, the Foundation was formed…
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A Team of Scientists Researching A Cure for Neurofibromatosis Type 1
