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…
Cures Within Reach is an organization that aims to help patients by repurposing research. They work towards getting treatments quickly and safely in an effort to aid those who need…
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…
Earlier this year, The Leukemia & Lymphoma Society hosted a presentation by Dr. Eunice Wang, Clinical Leukemia Service, Professor of Oncology, Roswell Park Comprehensive Cancer Center. Dr. Wang’s presentation…
If you're like me, your life has changed immensely over the past few months. COVID-19, a novel coronavirus with 8.39 million diagnoses worldwide, has kept us inside and changed…
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…
Older men who have a weak or irregular circadian rhythm — the body’s internal clock that controls the sleep-wake cycle — are approximately three-times more likely to develop Parkinson’s disease…
Antigen-specific T-cell therapy is an innovative treatment option which uses immune cells to combat cancer cells. AdventHealth is the very first in the world to use this treatment for patients…
According to Myasthenia Gravis News, CAAR T-cell therapy may be a viable treatment option for those with myasthenia gravis. Researchers from the University of Pennsylvania, Yale Medical School, and Cabaletta…
During this time of COVID-19, many people have some general information about COvID-19 including the knowledge that individuals over 65 years of age are at higher risk of contracting…
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…
Regeneron Pharmaceuticals recently announced that it is testing a medicine for the treatment and/or prevention of Covid-19. According to an article by CNN, the testing of its anti-SARS-CoV-2 antibody combination…
Regeneron Pharmaceuticals recently announced that it is testing a medicine for the treatment and/or prevention of Covid-19. According to an article by CNN, the testing of its anti-SARS-CoV-2 antibody combination…
Parkinson's News Today recently published an article featuring a study in Italy showing the benefits of deep brain stimulation in patients with late-stage Parkinson’s disease. The history of the subthalamic…
This year, thanks to COVID-19, the European Hematology Association (EHA) took their annual conference online - but that didn't prevent some intriguing new insights and ideas. In a press release,…
According to a recent article in PubMed, immune checkpoint inhibitors (ICIs) are the most commonly used type of cancer immunotherapy. ICIs have brought about a substantial improvement in the prognosis…
According to a recent article in Fiercepharma, vaccines based on new technology are being developed by AstraZeneca, Moderna, and Johnson & Johnson. The trials will be funded and conducted by…
According to an article in the Washington Post, a recent comment by a WHO official caused a furious debate and criticism of the organization. Maria Van Kerkhove, the head of…
by Danielle Bradshaw, In the Cloud Copy A common occurrence among children that have rapid-onset obesity with hypothalamic dysfunction, hypoventilation, and autonomic dysregulation or ROHHAD syndrome is that they’re also…
By Lauren Thayer from In The Cloud Copy Latent autoimmune diabetes in adults or LADA is a slow progressing form of autoimmune diabetes. Similar to type 1 diabetes, also an…
Over 2018 and 2019, BioCryst Pharmaceuticals conducted two surveys to better understand the needs and expectations of hereditary angioedema (HAE) patients. Overall, they found that despite progress in HAE therapies,…
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 the ASCO Post, researchers have discovered genomic characteristics of smoldering multiple myeloma that can tell one's risk of progression to multiple myeloma. Dr. Mark Bustoros and his team…
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…
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