When Carly Joseph first heard that her daughter Marissa had vomited at nursery, she wasn’t too concerned. After all, maybe Marissa just didn't feel great that day. But her worry…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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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