According to a story from The Del Mar Times, the Susan Poorman Blackie Ovarian Cancer Foundation (SPB Foundation) and the Clearity Foundation have recently partnered in order to facilitate a…
According to a story from express.co.uk, Elle Morris was only 11 years old when she died from a severe lung infection. Elle was born with cystic fibrosis, a serious genetic…
According to a story from jsonline.com, Tyler Trent, a Purdue University student who captured the hearts of thousands with his struggle against osteosarcoma, passed away at age 20 after his…
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,…
According to a story from BioPortfolio, the biotechnology company Tiziana Life Sciences plc recently announced encouraging results from an interim safety review of the company's Phase 2a clinical trial. This…
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…
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Dr. David Porteous, a General Practitioner in Bristol, England recently reported to the Bristol Cable, a community-run newspaper, that he does not agree with the extensive list of treatments being…
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…
According to a press release from Massachusetts-based biotech company Fulcrum Therapeutics, the company has successfully secured global commercialization rights of GlaxoSmithKline-developed experimental facioscapulohumeral muscular dystrophy (FSHD) drug losmapimod. Losmapimod, an…
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…
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Just one year ago David Chen, a forty-year-old policeman who lives in Toronto, was walking home after playing volleyball. According to a recent article in CBC Canada News, a…
There is a new show on television that showcases the experiences of patients and doctors at the Queen Elizabeth Hospital in Birmingham. On its first premiere, it showed the…
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…
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,…
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