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…
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…
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 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…
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).…
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…
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…
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…
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…
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…
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…
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…
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 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 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…
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…
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