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…
According to a story from BNN Bloomberg, Novartis AG, a drug company that is nearing the completion of the development of a potentially groundbreaking new gene therapy for spinal muscular…
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…
According to a story from Healio, a recent study has found that 58 percent of patients with the inflammatory disease ankylosing spondylitis will experience relapse within one year after stopping…
According to a story from finanzen.ch, the pharmaceutical company Eisai, Inc. announced that that company presented a total of four abstracts which were related to a Phase 3 clinical trial…
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…
According to a story from globenewswire.com, the biotechnology company Biogen recently released data from an open label study that was testing the company's spinal muscular atrophy drug nusinersen (marketed as…
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…
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According to an article in Biospace, the FDA is currently reviewing data from the Phase III STRIVE clinical trial for possible approval of Zolgensma as gene therapy for spinal…
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…
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…
According to a publication from the BBC, a novel new technology could help some with Parkinson's disease regain limited walking ability. However, unlike medical technology we often showcase on Patient…
According to a publication from Healio, recent breakthroughs in medical science have renewed hopes of finding a cure for the rare Cushing disease. Dr. Shlomo Melmed, dean and executive Vice…
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.…
According to a story from EurekAlert!, scientists have always sought to improve their understanding of the role that the environment and genes play in different diseases, as well as the…
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…
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…
According to a story from EurekAlert!, effective management of Parkinson's disease can be a serious challenge. Part of the reason that the management of the illness is so difficult is…
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