My name is Kelli Frederick, M.A., and I have lived with neurofibromatosis for my entire life. I have also undergone more than 50 facial surgeries. When you live with a…
As reported in the European Medical Journal, new research suggests that cherry angiomas occur more frequently and at an earlier age in people with neurofibromatosis type 1 (NF1), raising the…
The U.S. Food and Drug Administration (FDA) has approved Koselugo (selumetinib), an oral MEK inhibitor developed by Alexion, AstraZeneca Rare Disease, for adults with neurofibromatosis type 1 (NF1) who have…
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…
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…
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…
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…
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)…
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…
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…
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…
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).…
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…
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…
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…
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…
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…
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…
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.…
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…
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…
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…
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…
The Joint Global Neurofibromatosis Conference: Combining the 2018 Children's Tumor Foundation NF Conference, the 18th European Neurofibromatosis Meeting, and 2018 Schwannomatosis & NF2 State of the Art This event…
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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