In the United States, the FDA grants Orphan Drug designation to drugs or biologics that are intended to treat, diagnose, or prevent rare conditions. A rare condition is one…
For patients who were given a diagnosis of advanced, inoperable (unresectable) biliary tract cancer, an article appeared in Healio with some encouraging news. Gemcitabine and cisplatin drugs have…
Rare diseases are, by their nature, rare—and because of this, it can be difficult to spur drug development. The FDA worked to overcome this through the creation of the…
According to an article in GastroEndo News, first published in Clinical Oncology News, the U.S. Food and Drug Administration (FDA) recently approved a combination of durvalumab and gemcitabine plus…
In Europe, Orphan Drug designation is granted to therapies intended to treat, prevent, or diagnose a rare, life-threatening, or debilitating disease. For the purpose of this status, rare conditions…
Nelson Ambrogio had always looked up to his older brother, Daniel. He has many positive memories of growing up with him. They both shared a passion for soccer, and Nelson…
In late February 2022, biopharmaceutical company Trascenta Holding Limited ("Trascenta") provided an update on its clinical program for TST001, a monoclonal antibody therapy designed for those with locally advanced or…
According to a recent article, researchers have found that adding durvalumab to chemotherapy treatments results in prolonged survival rates in patients with advanced biliary tract cancer. Biliary Tract Cancer Biliary…
Silmitasertib, an investigative therapy for biliary tract cancer (BTC), has been awarded Orphan Drug Designation by the FDA. Researchers and the regulatory agency are hopeful that this therapy could become…
A Phase 3 clinical trial for advanced biliary tract cancer (BTC) has found that there is an improved overall survival rate when patients are given the immunotherapy durvalumab in combination…
Happy Friday! This week, author Tom Seaman asks: When it comes to anxiety, are you turning your Gizmo into a Gremlin? Also, in our new Compassion Corner series, we examine…
According to MPR, the FDA recently granted Orphan Drug designation to CPI-613 (devimistat), an investigational treatment designed for patients with biliary cancer. Altogether, "biliary cancer" can consist of bile…
In late May 2021, the FDA granted accelerated approval to Truseltiq (infigratinib). As reported by Cure, this therapy is designed for adult patients with unresectable locally advanced, previously treated, or…
A biological marker ("biomarker") is a measurable trait or characteristic that can tell researchers something about your body or health. For example, body mass index (BMI) is a common biomarker…
Just because we are staying home to avoid COVID-19 doesn't mean the medical field has stopped! In fact, many doctors are more determined than ever to improve patient outcomes.…
According to a story from globenewswire.com, the biopharmaceutical company Lexicon Pharmaceuticals has recently announced that it has completed its safety review of the initial cohort for its phase 2 clinical…
According to a publication from the Arizona BioIndustry Association, Dr. Rachna Shroff and a team of researchers from the Arizona Cancer Center are studying the use of nab-paclitaxel (a pancreatic…
According to a story from Healio, there are a number of recent reports of progress in the development of treatments for rare forms of liver disease. Many rare liver diseases…
According to a story from BioPortfolio, Imbrium Therapeutics and Mundipharma EDO GmbH recently announced that the US Food and Drug Administration (FDA) has awarded Orphan Drug designation to their experimental…
According to a story from EurekAlert!, the results from a recent Phase 2 clinical trial suggest that a two drug combination could be a useful approach for people with rare,…
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