According to a story from pm360online.com, the Orion Corporation, a pharmaceutical company based in Finland, has recently announced that it plans to continue its Phase 3 trial of levosimendan as…
According to a story from PR Newswire, The Lustgarten Foundation, which is the largest private organization that funds research on pancreatic cancer, has recently announced that opening of its fourth…
According to a story from Genetic Obesity News, the unusual case of a female Bardet-Biedl syndrome patient who also had endometrial cancer is causing researchers to wonder if the two…
According to a story from finanznachrichten.de, the Swedish drug company Calliditas Therapeutics has recently announced that the US Food and Drug Administration (FDA) has given one of the company's experimental drugs…
According to a story from Rare Disease Report, the US Food and Drug Administration (FDA) has announced its approval of a combination therapy from Janssen which consists of ibrutinib plus…
Mendelian Mendelian is a healthtech company in London whose primary goal is to help the National Health System (NHS) diagnose rare diseases faster. With 3 million people currently living with a…
Flesh eating bacteria is an infection that can have dire consequences. A Streptococcus, the most common of the flesh eating diseases, causes death in one third of all people…
Rett Syndrome Rett syndrome is a rare neurodevelopmental disorder. It affects females disproportionately, at a far greater rate than males. It causes cognitive, autonomic, and neurological dysfunction as well as a loss of…
Duchenne muscular dystrophy (DMD) has gained recent attention. New symptom management and gene therapy treatments are at the forefront of DMD research. DMD is a genetic disease characterized by progressive…
This is the story of Tom Dinwiddie, who was diagnosed with terminal cancer on April 2, 2012. This year, now age 69, he is officially six years cancer free. The Diagnosis…
According to a story from nbcwashington.com, Nikki Ferraro was diagnosed with a rare type of thyroid cancer when she was just seventeen years old. While she was able to survive…
According to a story from BioSpace, the biotechnology company REGENXBIO, Inc. recently announced that its experimental gene therapy candidate called RGX-181 has been granted Rare Pediatric Disease designation from the…
What is osteogenesis imperfecta? Osteogenesis imperfecta (OI) actually refers to a group of four distinct rare disorders- OI Type I, II, III, and IV. They're all characterized by brittle bones which…
Thanks to a chance encounter in a bike shed, two research groups at the Francis Crick Institute have discovered two distinct ways that pancreatic cancer tumors form and grow. The first research…
According to a story from independent.co.uk, organ transplants have been responsible for saving many lives. Organ transplants are often a critical part of treatment for a lot of rare diseases.…
According to a story from Reuters, a new website developed by the American Gastroenterological Association is meant to help women that have inflammatory bowel disease have safe and healthy pregnancies.…
According to a story from WebMD, the rare nervous system disorder chronic inflammatory demyelinating polyneuropathy is notorious for being difficult to diagnose. Many rare diseases can be challenging to identify.…
The Super Bowl is obviously a big deal for football fans - but it's also (for a lack of a better term) the SUPER BOWL of commercials. Over 100 million…
According to a story from Medical News Today, leaving ulcerative colitis (a form of inflammatory bowel disease) untreated could result in dire consequences for a patient. While ideally all patients…
A recent study, called TOPCAT, evaluated the effects of spironolactone as a treatment for individuals living with Chronic Kidney Disease (CKD) who are also diagnosed with heart failure with preserved ejection fraction,…
Familial Chylomicronemia Syndrome (FCS) Is a rare disease caused by malfunctioning lipoprotein lipase. This results in a buildup of triglycerides in the body's plasma. FCS can cause pancreatitis, memory loss, nerve…
According to a story from mirror.co.uk, a five-month-old girl named Maryam Malji was diagnosed with spinal muscular atrophy, a progressive, degenerative disease that is ultimately fatal without treatment. Her brother…
Time for Revision Like many things in this world, research for rare diseases such as Spinal Muscular Atrophy (SMA), Multiple Sclerosis, and Duchenne Muscular Dystrophy (DMD) has evolved substantially in recent years. This evolution…
AL Amlyoidosis is a rare disease which causes a buildup of harmful proteins (amyloids) in organs such as the kidneys and the heart. This accumulation causes organ damage as well as…
According to a story from GoodRx, hyperkalemia, which is a condition of elevated levels of potassium in the blood, can be a potentially life threatening situation. The condition can appear…
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