Historic Milestone Offers Relief from Relentless Burden of Glycogen Storage Disease Type Ia According to a recent press release from Ultragenyx, after decades of medical innovation and tireless advocacy from…
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…
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…
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…
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