Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
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…
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…
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…
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…
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.…
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),…
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…
Earlier this year, the MHRA in the United Kingdom expanded the license for Kaftrio, a treatment option for patients with cystic fibrosis (CF). When this expansion occurred, allowing for the…
Unfortunately, there are a number of racial and ethnic disparities in healthcare which position certain groups for poorer outcomes. For example, Black individuals often have lesser access to adequate medical…
When Isabel Blevins was just 16 years old, she caught the flu. While she recovered well at the time, her health seemingly went downhill after this incident. Suddenly, Isabel was…
Each year, there are an estimated 220,000 new cases of cryptococcal meningitis. Unfortunately, researchers hypothesize that this results in over 100,000 to 180,000 deaths; many of these are in those…
There are multiple factors which may play a role in the development of cerebral palsy, a neurological movement disorder. This condition can be caused by injuries to the brain at…
In 2018, Ethan Lambert was born. His parents, Kassondra and William, were thrilled with the new addition to their family. For the first year, Ethan seemed to be developing fairly…
In the past, certain research has suggested a relationship between the use of therapies like TNF inhibitors, tocilizumab, and tofacitinib, and a heightened risk of Alzheimer’s disease (AD) or dementia.…
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…
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”…
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…
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;…
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.…
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