Rare Disease Month is deeply personal to our family. Before Pruitt, our youngest son, we had never heard of Ornithine Transcarbamylase (OTC) Deficiency, nor did we truly understand how fragile…
In 2012, the FDA established its Rare Pediatric Disease designation initiative through the Food and Drug Administration Safety and Innovation Act. Rare Pediatric Disease designation is granted to drugs or…
Written by Venus Loreto In 2011 and 2016 I lost two children who were both 2 days old. My baby boy who was born in 2011 had an onset of…
In a recent press release, biopharmaceutical company Ultragenyx Pharmaceutical Inc. ("Ultragenyx") shared positive safety and efficacy data from Phase 1/2 clinical trials on gene therapy solutions for ornithine transcarbamylase…
In early December 2020, mRNA medicines company Arcturus Therapeutics Holdings Inc. ("Arcturus") announced that the company begun dosing participants within its Phase 1b clinical trial. Within the trial, researchers will…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Ornithine transcarbamylase (OTC) deficiency is a disorder that can cause comas, seizures, and death, making it necessary for those affected to seek treatment. Arcturus Therapeutics aims to give those with…
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Gene therapy is the treatment of disease by way of the transfer of genetic material into cells. According to a recent article in Labiotech, an EU publication, gene therapy…
According to a story from globenewswire.com, the messenger RNA therapeutics company Translate Bio recently released the latest updates in regards to two of its leading product candidates for the treatment…
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