Mastocytosis refers to an entire group of disorders. All of these conditions are characterized by a rapid production of mast cells (MCs), which are abnormal. This overproduction can occur in…
Rhabdomyosarcoma is a rare form of cancer that affects children. It was recently studied at St. Jude Children's Research Hospital, where a team of medical professionals examined cells that persist…
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…
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…
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…
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…
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…
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…
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…
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”)…
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),…
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…
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…
Understanding rare diseases is integral in treating them. In order to do so, research into the underlying causes and factors behind these conditions is needed. A recent study published in…
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…
Theravance Biopharma Inc. has just released results from their 2nd Phase 3 investigation of Ampreloxetine as a therapy for symptomatic neurogenic orthostatic hypotension (nOH). Symptomatic nOH NOH is a rare…
Dr. Christopher Austin, a National Institute of Health Director for Transitional Sciences, spoke at last year’s virtual conference honoring Rare Disease Day. According to an article in the NIH Record, Dr.…
Many of us are familiar with the famous story written by Roald Dahl titled Charlie and the Chocolate Factory; we know the tale of the golden ticket and Charlie's journey to…
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…
Korea began its use of effective therapies for idiopathic pulmonary arterial hypertension (IPAH) early in 2000. Idiopathic hypertension has no apparent cause. Yet there have been very few studies assessing…
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.…
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