
Transplant Operation Gives Teen With Wilson’s Disease a Second Chance
According to a story from the Hindustan Times, seventeen year old Yash Galpelli was born with Wilson's disease, a rare genetic disorder. For a long time, he had no idea…
According to a story from the Hindustan Times, seventeen year old Yash Galpelli was born with Wilson's disease, a rare genetic disorder. For a long time, he had no idea…
According to a story from MDedge, a review of patient data found that patients with osteomyelitis were more likely to need amputation within a two year period if they had…
According to a story from wect.com, local singer Dustin Chapman of Whiteville, NC has been struggling for years against a rare disease called achalasia, which affects the esophagus. He recently…
Ehlers-Danlos Syndrome Ehlers-Danlos syndrome (EDS) refers to a group of rare disorders which affect the connective tissues in the blood vessels, bones, skin, and other organs in the body. It's caused…
A Brazilian company known as Dr. Consulta is taking healthcare to a place only previously known by fast food chains. The company, founded in 2011, breaks the mold as a…
One of my better friends within the cystinosis community is Mika Covington. We met at a cystinosis event in 2016. I had read some of Mika’s entries posted in her…
We’ve all become a little more in tune to what’s good for our bodies, and what isn’t. Part of that is medical science finally being able to identify our bodily…
Multiple Myeloma Multiple Myeloma is a rare form of cancer that develops in the body's plasma cells. These cells are normally responsible for helping the body fight infection. The accumulation…
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 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 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…