On April 20, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
Dr. Alan Grant (portrayed by Sam Neill) began Jurassic Park with a distaste for computers and children, intent only on developing his paleontological prowess. By the end of the…
In a news release from mid-April 2023, clinical-stage biotechnology company Abivax SA shared that 2-year results were available from a Phase 2b open-label maintenance study evaluating obefazimod for individuals…
In a news release on the company’s website, biopharmaceutical company Applied Therapeutics, Inc. shared that positive data was available from the Phase 3 ACTION-Galactosemia Kids trial. Within the trial, researchers…
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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