In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. Do you know what makes a…
According to a story from Markets Insider, the biopharmaceutical company CStone Pharmaceuticals has announced that China's National Medical Products Administration (NMPA) has approved avapritinib (marketed as Ayvakit) as a treatment…
Rebecca Newsome lives with a rare form of cancer called gastrointestinal stromal tumors (GISTs). In this story, she talks about her experiences as a patient in a question-and-answer format. What…
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…
Recently, Blueprint Medicines Corporation ("Blueprint") shared that the European Commission (EC) approved marketing authorization of AYVAKYT (avapritinib). The therapy is designed to treat adult patients with PDGFRA D842V mutated gastrointestinal stromal…
According to an article from BioTech 365, data from the Phase 3 INVICTUS trial and Phase 1 dose-escalation study, which evaluated QINLOCK as a treatment for gastrointestinal stromal tumors, was…
Last week, biopharmaceutical company Deciphera Pharmaceuticals announced results from its Phase 1 clinical trial on QINLOCK (ripretinib). The study, published in the Journal of Clinical Oncology, tested QINLOCK as a potential…
According to ONCLive, ripretinib, designed to treat patients with advanced gastrointestinal stroll tumors (GIST), was given Priority Review status in China by the National Medical Products Administration. This status…
Each year, June 13th is celebrated as Gastrointestinal Stromal Tumors (GIST) Awareness Day. The ultimate goal of awareness days for rare diseases is to help inform both the general public…
Surgery has been found to often be the best option towards a cure for a cancerous tumor (resectable) but there are many other tumors that are diagnosed as unresectable…
According to a story from BioSpace, the US Food and Drug Administration (FDA) has recently approved avapritinib (marketed as AYVAKIT) as a treatment for gastrointestinal stromal tumors in cases when…
While rare disease affects people all around us, often the mainstream press . Which is why it's extra important when a news outlet as big as the Los Angeles Times…
According to a story from BioPortfolio, the biopharmaceutical company Deciphera Pharmaceuticals, Inc., recently announced the release of preliminary data from the company's Phase I clinical trial, which is still in…
July 13th was Gastrointestinal Stromal Tumors Awareness Day! And that "13" has extra relevance. 5,000 Americans are diagnosed with Gastrointestinal Stromal Tumors (GIST) each year. That’s 13 people diagnosed each…
July 13th is Gastrointestinal Stromal Tumors Awareness Day! And that "13" has extra relevance. 5,000 Americans are diagnosed with Gastrointestinal Stromal Tumors (GIST) each year. That’s 13 people diagnosed each day.…
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