In early November, global biotechnology company BioMarin Pharmaceutical Inc. ("BioMarin") announced the acceptance of its New Drug Application (NDA) for vosoritide. The investigational injectable treatment is designed to treat pediatric…
Later this week, the American Association for the Study of Liver Diseases (AASLD) will hold its virtual The Liver Meeting Digital Experience (TLMdX). Altogether, TLMdX will last from November 13-16.…
In late October, molecular information company Foundation Medicine, Inc. ("Foundation") shared that its diagnostic tool FoundationOne CDx was approved as a companion diagnostic for VITRAKVI (larotrectinib). While FoundationOne CDx will…
Pancreatic cancer is a rare but severe cancer, and it is often difficult to diagnose until it has advanced. When you combine this with a lack of knowledge and awareness…
Tuberous sclerosis complex (TSC) is a rare disease that, like many other rare diseases, is not yet fully understood by medical professionals. Because of this, it is very difficult to…
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…
Bryan and Bradford Manning recently gave an interview to the Associated Press. The brothers told the story of their vision loss as a result of a rare degenerative eye disease.…
According to a story from PR Newswire, a new collaboration between the Hairy Cell Leukemia Foundation (HCLF) and the Leukemia & Lymphoma Society (LLS) will aim to support critical research…
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