Tuberous Sclerosis Tuberous sclerosis complex (TSC) is a rare condition which results in the buildup of tumors in the skin, kidneys, brain, as well as other organs. Although the tumors are…
Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is an autoimmune condition which causes inflammation throughout the digestive tract. There are two main forms of the condition- Crohn's disease and the…
Happy Valentine's Day! Whether you're looking forward to spending tomorrow with a partner, or with friends and family, we hope you're having a good week. Today, we're highlighting an about…
Gene Editing What is gene editing? It's been a hot topic over the last few years, but basically the concept is this: instead of disabling a malfunctioning gene, researchers are…
According to a story from Pharmacy Times, the US Food and Drug Administration (FDA) have recently approved the first ever therapy for the rare disorder acquired thrombotic thrombocytopenic purpura (aTTP),…
The Importance of Speech The ability to speak gives individuals not only the opportunity to communicate with one another, it provides an outlet for expression, creativity, and connectedness. Unfortunately, that…
Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a rare, progressive, and fatal condition. It primarily affects males (1 out of every 3,600 to 6,000 male births). It's caused by…
Merkel Cell Carcinoma Merkel cell carcinoma is a rare and aggressive form of skin cancer. It's most often caused by the Merkel cell polyomavirus. However, it can also occur as…
According to a story from PR Newswire, the biopharmaceutical company Soligenix, Inc. recently announced that it has received a patent in the EU for the company's innate defense regulator drug…
Lambert-Eaton Myasthenic Syndrome Lambert-Eaton Myasthenic syndrome (LEMs) is a rare autoimmune disease. The body's immune system targets nerve cells meaning patients experience muscle weakness. Other symptoms of LEMs include difficulty walking…
According to a story from FOX31 Denver, Kelly Conley of Arvada, Colorado saw her circumstances change dramatically when she first began experiencing symptoms of Cogan's syndrome, a rare disease that…
According to a story from BioPortfolio, the biopharmaceutical company Acceleron Pharma Inc., recently announced the release of results from a Phase 2 study which tested the drug luspatercept as a…
According to a story from the Hindustan Times, seventeen year old Yash Galpelli was born with Wilson's disease, a rare genetic disorder. For a long time, he had no idea…
According to a story from MDedge, a review of patient data found that patients with osteomyelitis were more likely to need amputation within a two year period if they had…
According to a story from wect.com, local singer Dustin Chapman of Whiteville, NC has been struggling for years against a rare disease called achalasia, which affects the esophagus. He recently…
Emmanuel Mignot, who is the director of the Stanford Center for Sleep Sciences and Medicine has long believed that some onsets of Narcolepsy could be caused by a confused autoimmune response in…
Ehlers-Danlos Syndrome Ehlers-Danlos syndrome (EDS) refers to a group of rare disorders which affect the connective tissues in the blood vessels, bones, skin, and other organs in the body. It's caused…
A Brazilian company known as Dr. Consulta is taking healthcare to a place only previously known by fast food chains. The company, founded in 2011, breaks the mold as a…
Tristan Lederman's health started to take a turn at the beginning of 2017. Tristan lived at home with his family and depended on his family members and at-home caregivers…
Narcolepsy and Cataplexy Narcolepsy is a neurological condition that results in a malfunctioning sleep-wake cycle. It affects approximately 185,000 individuals in the United States alone. Some of the most common…
Pheochromocytoma and Paraganglioma Pheochromocytoma refers to a type of tumor that arises from chromaffin cells. They normally occur in the adrenal medulla. However, 10% of these tumors occur outside of this…
Hypereosinophilic syndrome Hypereosinophilic syndrome (HES) refers to a group of rare conditions characterized by high levels of eosinophils which result in organ damage. These eosinophils often affect the lungs, heart, skin,…
Multiple Myeloma Multiple Myeloma is a rare form of cancer that develops in the body's plasma cells. These cells are normally responsible for helping the body fight infection. The accumulation…
About Graft-Versus-Host-Disease (GvHD) Graft-Versus-Host-Disease (GvHD) occurs when a patient's body rejects cells which have been transplanted. For instance, it may develop following a stem cell transplant or a bone marrow transplant. The…
The Problem One of the greatest challenges in the investigation of nanoscale treatments for people with rare diseases is figuring out how to administer the therapy to the correct part…
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