According to a story from ANCA Vasculitis News, a recent review study has determined that patients living with ANCA vasculitis and other related diseases are at a higher than average…
According to a press release from HemoShear Therapeutics, the company has recently received official clearance from the US Food and Drug Administration (FDA) in regards to its Investigational New Drug…
According to a story from Parkinson's News Today, a recent study has found that a combination treatment of levodopa and carbidopa delivered to the intestines could be an effective treatment…
The Rare Diseases Clinical Research Network (RDCRN) has recently launched a survey aiming to assess the impact of COVID-19 on the rare disease patient community. This initiative is important as…
According to a story from BioSpace, the biopharmaceutical company Dicerna Therapeutics, Inc. has announced recently that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to…
Last week, I had the privilege of attending a webinar organized by Research!America titled Partnership, Progress, Pandemic: The Impact of COVID-19 on Medical Discovery. As implied in the title, the…
According to a story from Biotech 365, the biopharmaceutical company Deciphera Pharmaceuticals has recently announced that the company's drug ripretinib (marketed as QINLOCK™) has been approved by Health Canada. The…
June 23rd, 2020 will be recognized as Dravet Syndrome Awareness Day, and June is Dravet Syndrome Awareness Month. These events are dedicated to spreading awareness about the disorder among the…
“The allergist I have here in town thinks I am *overreacting*. You mean, overreacting when my neighbors next door are cooking their meat on the grill and when I walk…
According to a story from ajmc.com, a recent study found that a new combination treatment for systemic amyloid light-chain (AL) amyloidosis is more effective than the current standard treatment. The…
According to a story from globenewswire.com, the biotechnology company CytoDyn Inc. has recently announced the initiation of a phase 2 clinical trial. This clinical trial will be testing the company's…
June 19th is recognized as World Sickle Cell Day. This day is set aside to help spread awareness about sickle cell disease and its variants among the general public and…
According to a story from PR Newswire, the US Food and Drug Administration (FDA) has just approved a treatment for rare adult-onset Still's disease for the first time ever. The…
According to a story from Renal & Neurology News, a recent study has found that rituximab is superior to azathioprine therapy in preventing symptoms relapse in patients living with ANCA…
According to a story from Medpage Today, the US Food and Drug Administration has approved the drug lurbinectedin (marketed as Zepzelca), developed by Jazz Pharmaceuticals, as a treatment for metastatic…
According to a story from Buzzfeed News, the results of a recent trial held in the UK may have finally revealed a medication that can have a major positive impact…
In the geographically small island nation of New Zealand, there are estimated to be as many as 300,000 people who live with various rare diseases or disorders. Unfortunately, like in…
According to a story from BioPharma Dive, the drug company Sarepta Therapeutics is pushing forward with its plans to develop a gene therapy for limb-girdle muscular dystrophy (LGMD). The company…
June 17th is recognized as World CDKL5 Awareness Day and the month of June is considered CDKL5 Awareness Month. First organized by the International Foundation for CDKL5 Research (IFCR), the…
According to a story from Medical Xpress, a group of researchers from Penn State University have designed a unique clinical trial that aims to measure the impact of exercise on…
According to a story from interestingengineering.com, the capabilities of CRIPSR gene editing technology have been creating a buzz in the medical field for years now, with many seeing the technology…
According to a story from Scleroderma News, June is recognized as Scleroderma Awareness Month. While physical distancing as a result of the ongoing coronavirus/COVID-19 pandemic may keep some folks at…
According to a story from PR Newswire, Akcea Therapeutics, Inc. recently announced that the Portuguese Medicine Regulatory Authority (INFARMED) has announced its approval of reimbursement for inotersen (marketed as TEGSEDI®)…
June is recognized as Myasthenia Gravis Awareness Month. Primarily organized by the Myasthenia Gravis Foundation of America (MGFA), this event is focused on spreading awareness as well as boosting efforts…
According to a story from PR Newswire, CureDuchenne Ventures has recently announced the initiation of its CureDuchenne Ventures 2020 Pitch Contest. This marks the first time that the contest has…
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