Uveitis Uveitis is a rare condition in which the uvea of the eye becomes inflamed. The uvea is responsible for providing the majority of the blood to the retina and…
An Unexpected Emergency Kristin Hardy and her sister Margaret are no strangers to rare disease. Kristin has primary progressive multiple sclerosis (MS) and Margaret has relapsing-remitting multiple sclerosis. Margaret also…
After 20 agonizing years, a mother and father have finally received answers regarding their daughter's health. It shouldn't take this long. The Diagnosis Their daughter was diagnosed with two very…
Sarcoidosis Sarcoidosis is a rare inflammatory condition that causes granulomas to buildup within the organs. These can cause permanent organ damage. While the condition can affect various organs, and presents…
Doctor Alan Pestronk has identified a new and very rare autoimmune disease. He is now trying to spread the word about its existence to other doctors in an effort to…
Noonan Syndrome Noonan syndrome (NS) is a rare disorder that is typically diagnosed at birth. It affects the child's development resulting in stunted growth, abnormal facial features, and heart defects,…
Doctor Ashutosh Mangalam recently conducted a study examining the affect of Prevotella histicola, or P. histicola, a bacteria found in the human gut, as a treatment for multiple sclerosis (MS). Specifically, he…
Stevens-Johnson Syndrome Stevens-Johnson Syndrome (SJS), otherwise known as toxic epidermal necrolysis, is a rare disease caused by allergic reactions to common drugs or a reaction to an infection. Some of…
Researchers have recently uncovered two new rare diseases caused by the same mutation. This recent discovery is giving more families answers, and should hopefully begin to improve patient outcomes. The…
Mesothelioma Mesothelioma is a rare form of cancer most commonly caused by asbestos exposure. Unfortunately, after the time of exposure, it can take between 20 and 50 years for a…
Wiskott-Aldrich Syndrome Wiskott-Aldrich Syndrome (WAS) is a rare immune disorder which causes platelets in the blood to function abnormally. It also causes autoimmunity. Patients suffer from severe bleeding which can…
As the medical field continues to advance, treatments are becoming less and less "traditional" because researchers are uncovering unique ways to combat illnesses. Therapies range from pills, to surgery, to…
Stargardt Disease Stargardt disease is a rare disease which results in progressive vision loss. It is also referred to as juvenile macular degeneration. Most often, the disease presents during childhood.…
Primary Sclerosing Cholangitis (PSC) and Autoimmune Hepatitis (AIH) are both rare and chronic liver diseases. They cause the bile ducts in the liver to become inflamed, resulting in severe liver damage.…
Hypereosinophilic Syndrome Hypereosinophilic syndrome (HES) is a rare disease which causes high levels of white blood cells in the body. These cells are called eosinophils, and they're essential for proper…
In a world that is just beginning to make space for the open discussion of transgender individual's life experiences, there's still a huge gap in the conversation regarding healthcare. This…
There was a time when most children diagnosed with a rare condition did not live until adulthood. But now, with advances in medicine, many individuals are living long, full lives…
Within the pharmaceutical industry, we need 1) better policies for patients and 2) improved communication/education of the policies that do exist for patients and the rights that they have. Ultimately,…
The Problem Lysosomal storage disorders (LSDs) affect approximately one out of every 7,000 people. There are around 50 different types of LSDs. These include Fabry disease, Batten disease, Gaucher disease,…
Neurodegenerative Diseases There are a wide range of neurodegenerative diseases including Huntington's disease and Parkinson's disease. These conditions typically affect the older population. As the world's population ages, more people…
Background Novartis ended all of their gene therapy programs which were oncology related in 2016. Unfortunately, that meant an end to many programs that still had potential. This included cell…
Results from the largest United States study to date of Lambert-Eaton myasthenic syndrome (LEMS) have indicated that for the majority of patients, Firdapse is the most effective treatment option currently…
Researchers at GENFIT are excited about promising results from studies investigating a new potential therapy for PBC and NASH, two rare liver diseases. Primary Biliary Cholangitis Primary biliary cholangitis (PBC)…
When you’re diagnosed with a rare condition it can change the entire course of your life, at least at the beginning. But those living with diseases like these are fighters.…
Severe Combined Immunodeficiency Severe Combined Immunodeficiency (SCID) is a rare disease which causes patients to practically have no immune system at all. That means that for these patients, even a…
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