The main symptom of Kleine-Levin syndrome – excessive sleepiness (hypersomnolence) – is also a key characteristic of many other sleep disorders, such as narcolepsy. Additionally, there are currently no observed…
According to a recent press release, the Australian Human Research Ethics Committee (HREC) has granted approval to Claritas Pharmaceuticals' submission for R-107. Now, the company is free to move forward with…
Necroptosis, a type of regulated cell death, has been implicated in diseases such as inflammatory bowel disease, as well as neurodegenerative diseases. According to a recent article in News Medical…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. For the purpose of this status, “rare”…
International Fibrodysplasia Ossificans Progressiva (FOP) Awareness Day is recognized each year on April 23rd. This is a time for spreading awareness about this rare disorder among both the general public…
Eric is 35 years old and has been living with a chronic rare illness for around 20 years: membranous nephropathy. When he was a teen, he noticed that his legs…
According to a recent article in Businesswire, The Lancet has published data from the third phase of the Marigold trial. This clinical trial evaluated ganaxalone - marketed as ZTALMY - as a…
Many patients with sickle cell disease (SCD) experience “pain crises,” or periodic episodes of extreme pain. These pain crises occur when sickle-shaped red blood cells block the flow of blood…
According to a recent article, the results of a new study show that using recombinant human growth hormone to treat children with idiopathic short stature and growth hormone deficiency aids…
Rise is a nonprofit organization that gives support to the disabled. Its services include rides by Lyft, the popular ride-sharing company. The funds are then reimbursed by their riders’ Medicaid…
In patients with hereditary angioedema (HAE), the C1-inhibitor blood protein does not function properly, allowing bradykinin peptides to build up in the body. These then cause inflammation and fluid leakage;…
The VHL Alliance has just recognized Loyola Medicine as a new VHL Clinical Care Center. Von Hippel-Lindau (VHL) disease patients require multidisciplinary care for their complex and rare condition, and…
According to information from the National Institute of Health, one in nine elderly people in the U.S. will be affected by Alzheimer’s disease (AD). A recent article in the Globe Newswire…
Have you ever heard of acral lentiginous melanoma? This rare form of skin cancer typically develops on nail beds, on the palms of the hands, or soles of the feet.…
According to a recent article, researchers have created a new screening tool that will aid in discovering if patients with chronic idiopathic axonal polyneuropathy have the TRR mutation. Hereditary Transthyretin…
Ovarian cancer is notoriously hard to treat, ranking as the fifth leading cause of cancer deaths in women. Diagnosis often does not come until the disease has already progressed, making…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
April 21st is AML World Awareness Day! Acute myeloid leukemia (AML) is a rare cancer which affects people around the world. On awareness day, patients, caregivers, healthcare organizations, advocates, physicians,…
Every year almost half of the fourteen thousand patients who have received bone marrow or stem cell transplants will develop chronic graft-versus-host disease (cGVHD). Currently, there are no options…
Acute myeloid leukemia (AML) is a cancer of the bone marrow and blood. It spreads rapidly. Therefore precise and swift treatment is critical. In a recent interview with Medical Life…
Six years ago, researchers began developing single-domain antibodies (nanobodies) to help identify different species of Ebola; their work can be found published in ACS Infectious Diseases. Initially, the nanobodies were created to…
In 2020, Tyrese Dibba wanted to make a change for those who were Deaf and hard-of-hearing. Tyrese himself is Deaf and also has partial vision loss, resulting from his CHARGE…
In the past, there have been a few case reports and other studies which theorized a potential link between eosinophilic conditions, such as eosinophilic esophagitis (EoE), and Celiac disease, a…
According to a recent article, the biopharmaceutical company Amryt has announced a study looking at Mycapssa to treat patients with carcinoid symptoms as a result of neuroendocrine tumors (NET). Neuroendocrine…
According to a recent article, researchers at the WORLDSymposium revealed they are looking into using pentosan polysulfate sodium to treat Maroteaux-Lamy syndrome. MPS VI (Maroteaux-Lamy Syndrome) Maroteaux-Lamy syndrome is…
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