Neutropenia Neutropenia is a rare condition that affects approximately 1 in every 200,000 newborns. It results in a reduced number of neutrophils in the body. Neutrophils are white blood cells,…
According to a story from globenewswire.com, the biopharmaceutical company Lysogene and the drug developer Sarepta Therapeutics, Inc., have recently announced that the first patient has been dosed in a Phase…
Interstitial Lung Disease in Systemic Sclerosis Systemic sclerosis (SSc), sometimes referred to as systemic scleroderma, is a rare autoimmune condition. It results in the buildup of fibrosis in the skin…
Cryptococcosis Cryptococcosis is a disease caused by the Cryptococcus fungus. It can affect the lungs, the spinal cord, or the brain. When the brain is infected, the condition is called…
Hypoparathyroidism Hypoparathyroidism (HP) is caused by low levels of the parathyroid hormone (PTH). Most people develop this disease after thyroid surgery as a result of accidental damage/removal of the parathyroid…
According to a story from BioPortfolio, Takeda Pharmaceutical Company Limited recently announced that its drug ADCETRIS (brentuximab vedotin) has seen its marketing authorization extended in the European Union. The European…
Content Warning: The following article discusses sexual abuse. According to a story from Buzzfeed News, many former patients of an esteemed medical researcher named Reginald Archibald, who was affiliated with…
In order to begin clinical trials for an experimental new drug, pharmaceutical companies in the U.S. are required to submit an Investigational New Drug (IND) application to the FDA. They…
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),…
According to a story from pm360online.com, the Novartis company Advanced Accelerator Applications S.A. recently announced that Health Canada has issued approval for Lutathera™ as a treatment for gasteroenteropancreatic neuroendocrine tumors…
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…
According to a story from abc13.com, Washington state appears to be poised to become the third US state to ban dwarf tossing, a humiliating public spectacle used as entertainment in…
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…
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 northjersey.com, Adam Levitz's life was hanging in the balance just a few weeks ago. His liver, which had endured years of damage from the rare…
According to a story from pm360online.com, The National Brain Tumor Society (NBTS) recently awarded $750,000 to the Global Coalition for Adaptive Research. This cash is slated to go towards the…
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…
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,…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
