On December 9th, 2020, a special edition of "Behind the Mystery: Rare and Genetic" will air on Lifetime TV as part of the acclaimed morning program The Balancing Act. It…
According to a story from GlobeNewswire, the biotechnology company Galecto, Inc., has recently published the results from its phase 2a clinical trial. This study was testing the company's experimental therapy…
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…
According to a story from ANCA Vasculitis News, a recent study has determined that monitoring changes in autoantibodies targeting the PR3 protein is useful for predicting ANCA vasculitis remission after…
According to a press release from Inovio Pharmaceuticals, the biotechnology company has officially dosed the first patient in its phase 1/2 clinical trial, which will test the company's investigational therapeutic…
According to a story from Newswise, the ongoing coronavirus/COVID-19 pandemic has become the subject of wild speculation. In our divided nation, many people have chosen to hear what they want…
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…
This week Hallmark Movies and Mysteries premiered The Christmas Bow, which features Joy Perry, a woman with Charcot-Marie-Tooth disease, playing a woman with CMT. And while CMT has been known…
On November 19, 2020, Research!America hosted an informational webinar titled "From Nobel Winning Science to Next Generation Treatment: Tracing the Path of a Rare Disease Breakthrough using RNAi." This webinar focused…
According to an announcement from the FH Foundation, data from the FH Foundation’s CASCADE FH Registry, published in The Journal of Pediatrics, has determined that children living with familial hypercholesterolemia regularly…
According to a story from Newswise, a collaborative research team from the University of California San Diego School of Medicine and the Rady Children's Institute for Genomic Medicine will be…
According to a story from PR Newswire, The Autoimmune Registry, Inc. (ARI) has recently released its first ever autoimmune diseases list. This list includes a total of over 150 distinct…
According to a press release from the biopharmaceutical company Sanofi, the company's investigational treatment avalglucosidase alfa will have its Biologics License Application (BLA) evaluated by the US Food and 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…
According to a story from Fierce Pharma, the drug company Alnylam Pharmaceuticals has seen its investigational therapy lumasiran (marketed as Oxlumo) recently gain approval for use in the EU as…
According to a story from PR Newswire, the pharmaceutical and diagnostics company The Menarini Group recently announced that its drug tagraxofsup (marketed as Elzonris) has a received a positive opinion…
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…
According to a story from GlobeNewswire, the rare disease liver company Albireo Pharma, Inc., recently announced positive results from a phase 3 study testing the company's experimental drug odevixibat as…
According to a press release from the biopharmaceutical company Sanofi, the Biologics License Application (BLA) for its drug avalglucosidase alfa has been accepted by the US Food and Drug Administration…
According to a story from Charcot-Marie-Tooth News, patients living with Charcot-Marie-Tooth disease have reported in a recent survey that their symptoms of pain and decline in mobility increased during the…
According to a story from Newswise, the genome editing company Intellia Therapeutics has recently announced that the first patient has been dosed in its phase 1 trial, which will test…
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…
According to a story from pharmalive.com, the drug company Calliditas Therapeutics will plan to seek approval for its therapy Nefecon from the US Food and Drug Administration (FDA). This approval…
According to a story from Cancer Network, the European Commission has recently approved a two-part combination treatment for advanced, homologous recombination deficiency (HRD)-positive ovarian cancer. This first-line maintenance treatment consists…
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