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…
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…
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…
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.…
Mesothelioma is a cancer which occurs in the thin layers of tissue (mesothelium) which surround most of the body's internal organs. It's often caused by asbestos exposure. However, symptoms can take…
Sickle cell disease (SCD) is a rare blood condition. It causes sickling in the red blood cells which results in the blockage of blood vessels and capillaries. The sickling process…
Ionis Pharmaceuticals and Roche have been collaborating on a drug for Huntington's Disease which will treat the root cause of the condition. Currently, there are no such approved treatments, meaning…
While juvenile idiopathic arthritis remains to be the most common rheumatic disease in children, researchers have long been stumped at its caused. The best prediction was that it originated as…
Non-tuberculous mycobacterial (NTM) infections are caused by bacteria found in the environment. In healthy individuals, these bacteria normally cause no harm. However, for those who have a compromised immune system or who…
Notable Labs Notable Labs was created after founder, Matt De Silva, felt he was left in a helpless point, following a year of research on treatment options for his father's Glioblastoma…
Systemic Sclerosis (SSc) is a rare disease that causes the skin to thicken and form into scar tissue. It can affect various parts of the body including the gastrointestinal track,…
Epidermolysis Bullosa Epidermolysis Bullosa (EB) refers to a group of rare conditions which are characterized by fragile skin. Basically, the disease is caused by separation between the layers of the skin.…
The History of West Nile Virus The West Nile Virus (WNV) was named for the location where it was originally found- the West Nile district in Uganda. It was discovered…
The most common treatment for someone who is discovered to have early stage kidney cancer is surgery. The patient's tumors and a part of their kidneys are removed through a…
Sarcoidosis is a rare condition which causes inflammatory cells to build up in various organs throughout the body. For 90% of people with a sarcoidosis diagnoses, the lungs are one…
The State of Cancer in the United States A recent report posted by the American Cancer Society has indicated that cancer rates in the United States have been continuously falling over…
Autoimmune hepatitis (AIH) is a rare condition which causes inflammation in the liver. It can lead to cirrhosis and ultimately, liver failure. The typical treatment for patients with the disease includes…
We've made a lot of progress in cancer research in recent years. Many novel therapies have been developed for illnesses previously considered untreatable. However, as new treatments have been discovered,…
Danon disease is a rare condition caused by a mutated LAMP-2 gene. This gene has three different forms- LAMP-2A, 2B, and 2C. Depending in which variant is mutated, individuals with…
aTTP aTTP, or acquired thrombotic thrombocytopenia purpura, is an immune condition which results in severe Thrombocytopenia. Thrombocytopenia means the body doesn't have enough platelets in the blood. In aTTP, it's caused…
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