Neurofibromatosis Type 1: A Mother’s Struggle Living with Thousands of Tumors
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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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In 10 Years, “Links for Lauren” Has Raised $500K for Neurofibromatosis
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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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May is Neurofibromatosis Awareness Month: Spreading Rare Disease 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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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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NFX-179 Granted Orphan Drug Status for Cutaneous NF1
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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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Study of the Week: New Genetic Predisposition Variants in Rhabdomyosarcoma
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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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ICYMI: First Drug to be Approved for Neurofibromatosis Type 1 and Plexiform Neurofibromas (NF1-PN)
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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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FDA Approves a Treatment for Neurofibromatosis Type 1 for the First Time Ever
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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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Experimental Treatment for Neurofibromatosis Type 1 Earns Orphan Drug Designation in the EU
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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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Children’s National Health System Researchers Receive Grants to Develop High-Tech Diagnostic Aids
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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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Potential Treatment for Neurofibromatosis Type 1 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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New Potential Treatment for Neurofibromatosis Type 1 Given Breakthrough Therapy Designation

A new medication called selumetinib has just received Breakthrough Therapy designation by the FDA for pediatric patients diagnosed with neurofibromatosis type 1 (NF1) or inoperable pleciform neurofibromas. This therapy has…

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New Open Data Portal Will Improve Neurofibromatosis Research
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New Open Data Portal Will Improve Neurofibromatosis Research

According to a story from EurekAlert!, a collaboration of Sage Bionetworks, The Children's Tumor Foundation (CTF), and the Neurofibromatosis Therapeutic Acceleration Program (NTAP) have announced the initiation of the very…

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“Many Faces of NF”: Artist Highlights Tumor-Causing Genetic Disorder
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“Many Faces of NF”: Artist Highlights Tumor-Causing Genetic Disorder

If you haven't heard of neurofibromatosis (NF), you’re in surprisingly good company. For most of her life, Rachel Mindrup, an artist, illustrator, and Resident Assistant Professor at Creighton University, who…

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