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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MEK Inhibitor Successfully Treats Young Patients’ Neurofibromatosis Tumors
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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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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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Experimental Neurofibromatosis Type 1 Therapy Earns Fast Track Designation
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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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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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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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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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Could Gene Editing Pigs Be the Future of Treating Rare Diseases?

The Atlantic featured this incredible story about the possibilities of using genetically altered piglets as avatars for children with genetic diseases. Furthermore, the article delves into the personal story of one…

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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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Highlights from RAREfest: Cambridge’s Rare Disease Festival

This weekend Patient Worthy attended RAREfest, an event being held in the UK by the Cambridge Rare Disease Network to raise awareness and promote discussion around rare disease issues. The…

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Experimental Treatment for NF-1 Gets Orphan Drug Designation
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Experimental Treatment for NF-1 Gets Orphan Drug Designation

According to a story from BioPortfolio, the biopharmaceutical company SpringWorks Therapeutics recently announced that the US Food and Drug Administration (FDA) has given the company's experimental product PD-0325901 Orphan Drug…

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Researchers Are Trying to Understand the Link Between Specific Genetic Variants and Symptoms in Neurofibromatosis Type 1
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Researchers Are Trying to Understand the Link Between Specific Genetic Variants and Symptoms in Neurofibromatosis Type 1

An international study led by researchers at the University of Alabama Birmingham has investigated the link between a specific genetic mutation underlying neurofibromatosis type 1, and the symptoms patients with…

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Orphan Drug Designation Has Been Awarded to Selumetinib for the Treatment of Neurofibromatosis Type 1
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Orphan Drug Designation Has Been Awarded to Selumetinib for the Treatment of Neurofibromatosis Type 1

The European Medicines Agency has awarded Orphan Drug designation to selumetinib for the treatment of neurofibromatosis type 1. You can read the source press release here, on Merck’s website. About…

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The First Double Face Transplant Has Just Been Performed on a Neurofibromatosis Patient in France
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The First Double Face Transplant Has Just Been Performed on a Neurofibromatosis Patient in France

A man in France has been the first person to ever receive two face transplants, reports the BBC. After Jérôme Hamon’s first transplant was rejected he underwent a second transplant…

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This Treatment for Neurofibromatosis Type 1 Just Got Orphan Drug Designation
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This Treatment for Neurofibromatosis Type 1 Just Got Orphan Drug Designation

According to a story from Business Wire, selumetinib, a drug in development for the treatment for neurofibromatosis type 1, was recently granted Orphan Drug Designation by the Food and Drug…

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