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…
Living with a rare disease is difficult for so many reasons. One of the lesser talked about struggles of living with a rare condition is the fact that so few…
Understanding which patients are at a higher risk of poorer outcomes is essential to provide them with the best treatment. That's why so many researchers focus on signs or markers…
PNH Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease where blood cells break apart and cause the contents to circulate freely within the bloodstream. This condition is caused by a…
Many father-son duos are close, but this pairing - reported on in an article from CBS58 - shares a special bond that was strengthen by the shared experience of a…
Colette Romero's son is diagnosed with eosinophilic esophagitis (EoE), a rare disease. She discusses how, among the challenges that come with this condition, education has presented extra difficulties. Education and…
Lysogene has just announced a new study has been published in the journal EMBO Molecular Medicine which demonstrates that DGKk is able to produce long-term rescue of fragile X syndrome (FXS).…
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…
Only a few hundred cases of alveolar capillary dysplasia with misalignment of pulmonary veins (ACDMPV) have ever been identified or recorded. While there could, arguably, be more cases – especially…
Many drugs, whether they are indicated for common or rare diseases, come at very high prices. Zynteglo, an investigational gene therapy for beta thalassemia, is most likely to stick to…
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