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.
On the first Sunday in May, Cincinnati, OH hosts the Flying Pig Marathon – its 26.2 mile race. This year marked the 24th annual event. But for 17-year-old Diego Ramirez,…
Clinical trials are an important tool within the healthcare realm. These trials assist in developing deeper understandings of diseases, assessing and discovering treatment options, and learning new ways of identification…
The Food and Drug Administration (FDA) has four distinct approaches to expedite the availability of therapies for rare, severe, or life-threatening illnesses: Priority Review, Breakthrough Therapy, Fast Track, and Accelerated…
For the last 52 years, individuals with Wilson disease have been relying, largely, on the same treatment options. These options, designed to remove copper from the body, include Cuprimine (penicillamine).…
In Europe, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare, life-threatening, or chronically debilitating conditions. For the European Union (EU), a “rare”…
Currently, there is no way to determine when a relapse will occur in relapsing and remitting multiple sclerosis (MS), one of the two main forms of MS. However, this may…
Dr. Omar P. Haqqani, MD FACS is currently the Chief of Vascular and Endovascular Surgery at the Vascular Health Clinics. He has a keen interest in understanding and addressing vascular…
It’s finally May – and you know what that means! That’s right, this month is Huntington’s disease (HD) Awareness Month. The theme for this month’s HD Awareness Month is #LetsTalkAboutHD.…
Rare Pediatric Disease designation is granted by the FDA to drugs or biologics intended to treat life-threatening diseases affecting individuals aged 18 or younger. In this case, rare conditions are…
When Krista Webb was just two years old, doctors determined that she had some level of hearing loss. Her family was surprised about this; nobody else in her family, and…
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…
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