deCODE Genetics, which is a research subsidiary of Amgen genomics based in Iceland, has recently created an extremely detailed genetic map. They wanted to uncover more about the human genetic…
According to a story from Renal & Urology News, a recent study found that monitoring of albuminuria, a condition in which the protein albumin appears in the urine, can help…
What is Duchenne Muscular Dystrophy? Duchenne muscular dystrophy (DMD) is a rare genetic condition that is ultimately fatal. It causes muscles to weaken and usually results in heart failure before age…
There is a high unmet need to find more effective treatments for liver cancer. Currently, treatment options for patients often only increase life expectancy by 3 to 4 months. An accidental…
According to a story from The People's Pharmacy, there are an increasing number of worrying stories and incidents that are calling into question the US Food and Drug Administration (FDA)'s…
Hope everyone's week is going well! Today, we're highlighting an article discussing the difficulties of securing a CIDP diagnosis, followed by an update on a Niemann-Pick Type C treatment. Next,…
According to a story from pm360online.com, the life sciences company Solid Biosciences has released some preliminary findings from its Phase 1/2 clinical trial of SGT-001. This investigational product is in…
According to a story from the New York Times, a 50 year old Houston man had seen his health decline rapidly in a little over a year. After declining from…
According to a story from Scientific American, the World Health Organization (WHO) recently decided to strike the diagnosis congenital Lyme disease from its International Classification of Diseases (ICD). The document is…
According to a story from philly.com, it was August 2018 when Lisa Semple of Cherry Hill was first diagnosed with breast cancer. Unfortunately, her breast cancer had reached stage four,…
According to a story from Science Daily, an experimental therapy for cancer could shut down tumor cells using an innovative mechanism that disrupts their circadian rhythm functions. A concept study…
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…
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