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…
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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