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 Reuters, a new website developed by the American Gastroenterological Association is meant to help women that have inflammatory bowel disease have safe and healthy pregnancies.…
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.…
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 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…
AL Amlyoidosis is a rare disease which causes a buildup of harmful proteins (amyloids) in organs such as the kidneys and the heart. This accumulation causes organ damage as well as…
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…
According to a story from arkansasonline.com, Rep. Julie Mayberry R-Hensley of the Arkansas General Assembly is sponsoring House Bill 1074. This legislation proposes adding the rare and potentially fatal genetic…
According to a publication from Breastcancer.org, black women are more likely than others to develop triple-negative breast cancer at some point in their lives. A study published by the journal…
Progress Report Debuts ASCO’s Research Priorities for the Cancer Community, Emphasizes Impact of Federal Funding FOR IMMEDIATE RELEASE January 31, 2019 Ashley Yum 571-483-1376 [email protected] ALEXANDRIA, Va. — Over the…
We've all seen the commercials - especially during the holiday season - for home DNA kits like 23andMe and Ancestry DNA, where a saliva sample can unlock your family heritage.…
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…
According to a story from the Tenterfield Star, a fundraiser was recently held for Tommy Johnston, a one year old boy who was diagnosed with the rare Niemann-Pick disease type…
According to a story from The New York Times, a new wave of research is starting to reveal the close connection between the human gut microbiome and the brain. Some…
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…
February 2nd is Neuroblastoma Awareness Day! It's important to know more about this rare cancer, since knowledge and familiarity are often the engines of progress. What is Neuroblastoma? Neuroblastoma is a…
According to a publication from PR Newswire, CloudMedx, a technology company from Palo Alto, is touting impressive results following a simulated exam pitting human doctors against their "deep learning" Clinical…
There is a great unmet medical need for patients living with metabolic disorders. There are few approved treatments for these conditions and many are fatal. These diseases include phenylketonuria, homocystinuria, tyrosinemia, and maple syrup…
Rare disease families deal with a lot. The last thing they should be forced to have on their mind is how to get access to care. Unfortunately, for many parents…
A recent survey conducted by researchers at the NYU School of Medicine has indicated that oncologists know much less about the unique cancer needs of the LGBTQ population than they thought.…
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