Pheochromocytoma and Paraganglioma Pheochromocytoma refers to a type of tumor that arises from chromaffin cells. They normally occur in the adrenal medulla. However, 10% of these tumors occur outside of this…
Hypereosinophilic syndrome Hypereosinophilic syndrome (HES) refers to a group of rare conditions characterized by high levels of eosinophils which result in organ damage. These eosinophils often affect the lungs, heart, skin,…
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…
About Graft-Versus-Host-Disease (GvHD) Graft-Versus-Host-Disease (GvHD) occurs when a patient's body rejects cells which have been transplanted. For instance, it may develop following a stem cell transplant or a bone marrow transplant. The…
The Problem One of the greatest challenges in the investigation of nanoscale treatments for people with rare diseases is figuring out how to administer the therapy to the correct part…
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…
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 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 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…
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…
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 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.…
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…
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…
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…
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…
Niemann-Pick Disease is a genetic lysosomal storage disease. Lipids accumulate in the lysosomes of cells, causing debilitating symptoms. There are three forms of the disease- A, B, and C. Each…
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