Prednisone is one of the more common corticosteroids prescribed to individuals with lupus to help reduce inflammation and decrease symptom burden. Corticosteroids are also prescribed for a number of other…
On April 28, 2022, the National Organization for Rare Disorders (NORD) and the Rare Disease Diversity Coalition hosted a webinar program titled "Walk in Our Shoes: The Experience of Rare…
It is no secret that there is an inequity when it comes to race and medical research. More often than not, research does not take all races and ethnicities into…
Don't forget to check out Part 1 of our interview with Katheron Intson, where we discussed her background, her research experience in GRIN1, and the reasons behind why she developed…
In a news release from May 3rd, 2022, rare disease and cancer therapeutic development company DepYmed Inc. shared that its lead PTP1B inhibitor, designed to treat Rett syndrome, was granted…
According to a recent article in Genetic Engineering and Biology News, Washington University and University of Hawaii researchers have uncovered a possible drug development candidate in soil bacterium called…
Acromegaly and gigantism are two disorders that share a main characteristic: abnormal growth. Because of this similarity, many people confuse one for the other. However, these are two distinct, separate…
The Fatty Liver Foundation (FLF) has just announced a new national survey which will document the experiences and needs of individuals living with non-alcoholic fatty liver disease (NAFLD) and nonalcoholic…
Necrotizing enterocolitis is a serious and potentially fatal intestinal disease which most often affects preterm infants. For many years, researchers have hypothesized that gut health – and in particular, the…
Compiling data, especially rare disease data, can be extremely helpful for research purposes and for doctors treating patients with that condition. Recently, the International Gaucher Alliance and Cerner Enviza have teamed…
According to a recent article, although some perspiration is normal to have, if you are having regular episodes of excessive sweating it could be the sign of something more serious.…
Katheron Intson is a passionate scientist; she likes understanding how and why things work the way that they do. So when Katheron’s friend reached out about a mysterious health problem,…
Endemic in areas of Africa, southeast Europe, Central Asia, and the Middle East, Crimean-Congo hemorrhagic fever is an often-fatal viral illness which can cause severe issues. Researchers estimate that this…
A recent study published in Science Translational Medicine has investigated a possible factor of the development of fetuses born to mothers with neuromyelitis optica spectrum disorder (NMOSD). Specifically, it looked…
A parent never wants their child to endure a medical issue, much less one as serious as bacterial meningitis. Unfortunately, Jay Aston - a singer from Bucks Fizz - has…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. Rare diseases are those affecting fewer than 200,000 Americans.…
Earlier this month, I wrote about how efzitimod, a therapy for systemic sclerosis, received Orphan Drug designation from the FDA. Now, another therapy – this one from Gesynta Pharma (“Gesynta”)…
Growing up, Tayla Clement never saw anybody that looked like her in the media she engaged with. You see, Tayla has Moebius syndrome, a rare congenital neurological disorder characterized by…
Christian Maugee and Shandra Trantham met as undergraduates at the University of South Florida (USF) and immediately clicked. Their first chance meeting was in the disability office. It was here…
When J.B. Lacombe's son was born, he showed signs of jaundice. Jaundice, caused by extra bilirubin in the blood, results in the yellowing of the skin, eyes, and mucous membranes.…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Nobelpharma has just announced the FDA approval of sirolimus .2% gel (called Hyftor). It is now the first approved, topical therapy for facial angiofibroma associated with the rare condition tuberous…
Clinical trials are important in developing a better understanding of certain diseases or conditions, as well as how to treat them. According to HCP Live, researchers evaluated PRX-102 (pegunigalsidase alfa),…
When we are faced with a limiting health issue where performing “simple” everyday activities becomes a challenge, our self-confidence and self-worth can dwindle. This can happen with any health condition…
It's funny, I have to laugh every time someone asks me if I am pursuing a degree in Genetics because of my own genetic disease. Actually no. I knew I…
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