According to a story from Market Watch, the biopharmaceutical company Ultragenyx Pharmaceutical, Inc. recently announced that it was given Orphan Drug designation from both the US Food and Drug Administration…
Prior to this year, there were no reported cases of monkeypox in the United States since 2003. This all changed when a Texas man brought the rare disease back with…
According to a relatively recent news release from biopharmaceutical company Dicerna Pharmaceuticals, Inc. ("Dicerna"), interim data is now available from a Phase 1 clinical trial evaluating belcesiran for patients with…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
According to a July 27 news release from biopharmaceutical company Day One Biopharmaceuticals ("Day One"), the company's therapeutic option DAY101 received Rare Pediatric Disease designation within the United States. Altogether,…
In a news release from July 21, 2021, biotechnology company Genentech shared that its therapy Venclexta (venetoclax), in conjunction with azacitidine, received Breakthrough Therapy designation from the FDA. Overall, the…
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…
Sometimes, clinical trials do not go the exact way that is expected. According to Targeted Oncology, this is what happened in a Phase 1b clinical trial evaluating RVU120 for…
A survey of oncologists has found that more education is clearly needed regarding cholangiocarcinoma. Specifically, more education would be beneficial regarding testing and targeting the mutations which cause the condition…
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