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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RDMD Gets $14 Million in Financing, Partners with UCB to Research Progressive Supranuclear Palsy
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RDMD Gets $14 Million in Financing, Partners with UCB to Research Progressive Supranuclear Palsy

  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…

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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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Planting Love Around the World: A Young Patient with Neurofibromatosis Works to Reduce COVID-19 Anxiety
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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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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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Even in Death, This Glioblastoma Patient’s Selfless Lego Donation Drive Touches Thousands of Lives
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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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Scientists Are Using Pigs to Develop a More Useful Animal Model of Neurofibromatosis Type 1
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Scientists Are Using Pigs to Develop a More Useful Animal Model of Neurofibromatosis Type 1

According to a story from wearegreenbay.com, a team of scientists associated with the University of Wisconsin, Madison are undertaking a project that will help improve the effectiveness of research related…

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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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Family Support: Twin Sister Carries Twins for Neurofibromatosis Type 2 Patient
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Family Support: Twin Sister Carries Twins for Neurofibromatosis Type 2 Patient

According to a story from The Columbian, twins Jill Noe and Whitney Bliesner had developed a deep bond during their childhood days growing up in Oregon. However, they did not…

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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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After Neurofibromatosis Type 2 Prevented her From Getting Pregnant, her Twin Offered to Serve as a Surrogate
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After Neurofibromatosis Type 2 Prevented her From Getting Pregnant, her Twin Offered to Serve as a Surrogate

According to a story from KTVZ, Jill Noe and Whitney Bliesner have always shared a special bond. They are twins, now 34 years old. Despite their closeness, they do share…

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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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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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A Man Who Set Up a Healthcare Company After Being Diagnosed with Neurofibromatosis Has Secured $3 Million Funding

  The healthcare technology company RDMD was founded by Onno Faber, who has himself been diagnosed with a rare disorder. RDMD, which describes itself as “dedicated to accelerating research” with…

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