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…
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