Ultragenyx Pharmaceutical, a biotechnology company developing treatments for rare diseases, has received a setback from the U.S. Food and Drug Administration (FDA) regarding its gene therapy candidate, DTX401. According to…
In January of this year, a University of Connecticut (UConn) team administered the world’s first infusion of a new gene therapy as part of a long-awaited clinical trial. According to…
Written By Katie Ward I am not one who likes to bring attention to themselves, and you won’t ever find me talking about my struggles, issues, or anything negative. I…
In a news release from June 2, 2021, biopharmaceutical company Soleno Therapeutics, Inc. ("Soleno") shared an exciting update. The company's investigational treatment, DCCR (diazoxide choline) Extended-Release tablets, received Orphan Drug…
Happy Friday! This week we have details about an investigative therapy for Duchenne Muscular Dystrophy that has entered a phase 3 clinical trial. Also, have you ever heard of Glycogen…
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…
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…
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…
When your child is constantly at risk of hypoglycemia and their health and life is threatened in the case of any major problem, you have no other choice but to…
According to a source article that can be found here on Ultragenyx’s website, the first patient has been dosed in a trial of an investigational gene therapy called DTX401 that…
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