As reported in Charcot Marie Tooth News; researchers have devised a new measure of quality of life for youth with Charcot-Marie-Tooth disease. While of course, researchers could assess the youth’s…
In a recent press release, biopharmaceutical company Mirum Pharmaceuticals, Inc. ("Mirum") shared that the company finished its rolling New Drug Application (NDA) submission. The NDA focuses around maralixibat, an investigational…
GeneDx, an OPKO Health company, has recently launched several genetic tests. Within this group of tests are repeat expansion analysis for Friedreich ataxia, spinocerebellar ataxia (SCA), and other forms of…
Okay, I'll admit it -- I'm a bit of a reality TV junkie. So I have been watching Cheyenne Floyd's journey since her initial entrance on Are You The One? Season 3…
Charlie Fry is a three-year-old from Kansas City living with SLC6A1. His family is very active in the SLC6A1 community, even starting a nonprofit titled "A Cure for Charlie." They…
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…
Recently, biopharmaceutical company Soligenix, Inc. shared that it had been granted a patent (#16102842.8) in Hong Kong for the use of synthetic hypericin to treat patients with cutaneous T-cell lymphoma…
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