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 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 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 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…
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…
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.…
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 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…
According to a story from labiotech.eu, the biotechnology company Orphazyme has been developing an experimental therapy for Niemann-Pick disease type C. In a recent Phase 2/3 clinical trial, the investigational…
We've all seen the commercials - especially during the holiday season - for home DNA kits like 23andMe and Ancestry DNA, where a saliva sample can unlock your family heritage.…
Hereditary angioedema (HAE) is a rare condition which causes severe swelling in various areas of the body. Takhzyro has been approved as a treatment for this disease. The drug was created…
According to a story from the Tenterfield Star, a fundraiser was recently held for Tommy Johnston, a one year old boy who was diagnosed with the rare Niemann-Pick disease type…
February 2nd is Neuroblastoma Awareness Day! It's important to know more about this rare cancer, since knowledge and familiarity are often the engines of progress. What is Neuroblastoma? Neuroblastoma is a…
According to a publication from PR Newswire, CloudMedx, a technology company from Palo Alto, is touting impressive results following a simulated exam pitting human doctors against their "deep learning" Clinical…
Rare disease families deal with a lot. The last thing they should be forced to have on their mind is how to get access to care. Unfortunately, for many parents…
According to a story from BBC, it is becoming increasingly clear that a lot of the common phrasing and language that we use to talk about cancer and cancer patients…
Sickle cell disease (SCD) is a rare blood condition. It causes sickling in the red blood cells which results in the blockage of blood vessels and capillaries. The sickling process…
Non-tuberculous mycobacterial (NTM) infections are caused by bacteria found in the environment. In healthy individuals, these bacteria normally cause no harm. However, for those who have a compromised immune system or who…
Happy Wednesday! While it's felt like January was going to last forever, we're almost through the first month of 2019. As we gear up for February, we want to reflect…
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