It can be extremely difficult to incentivize drug development for rare diseases or conditions. To combat this, decades ago, the FDA created the Orphan Drug Act. Now, Orphan Drug designation…
Before you read on, make sure to check out Part 1 of our interview where we discuss what SLC6A1 is and Aubrey's diagnostic journey. Today, we'll be discussing what it's like to…
According to a late October 2022 news release from pharmaceutical company KalVista Pharmaceuticals, Inc. (“KalVista”), the company released positive data from a Phase 1 study evaluating oral sebetralstat for…
In recent years, treatment options for cystic fibrosis, a rare disease, have increased significantly in number and in effectiveness. However, many of the most effective treatments, developed by Vertex Pharmaceuticals,…
Before you read on, make sure to check out Part 1 of our interview. In Part 1, we discussed Dr. Sumeray's background, what hypoparathyroidism is, and its symptoms and treatments. Today, we're…
Both Libtayo (cemiplimab) and Keytruda (pembrolizumab) have been shown to be beneficial treatment options for patients with locally advanced or metastatic cutaneous squamous cell carcinoma (cSCC). But could Opdivo (nivolumab)…
When Erika Vandenberg learned that her daughter Aubrey had a rare genetic disorder called SLC6A1, her first two reactions were fear and relief. Relief because they finally had a name…
By Randall and Emma Barker Randall and Emma Barker have the unique experience of being a father and daughter both navigating life with Type 1 diabetes (T1D). With over two…
Atlanta, Georgia’s Linseigh Green received a diagnosis of necrotizing enterocolitis (NEC) when she was born. She was sent to the hospital’s NICU only two weeks after her birth. Linseigh’s…
When it comes to drug development and medical research, particularly within the rare disease sphere, it is incredibly important for patients to feel involved in the process. After all, patients…
The XV International Conference on Rare Diseases-D'Genes was streamed in sixteen countries in April 2022. Juan Carrión, D’Genes President, announced that the theme for 2022 is Equity and Rights…
Researchers have known about EGCG for decades. It is a plant compound called catechin found in green tea that may prevent disease and protect cells from damage. According to…
Newborn screening is a public health program in which infants are screened for various metabolic, genetic, and developmental disorders shortly after birth. Through newborn screening, doctors may identify potential disorders…
Currently, there are a number of therapies which can be used to treat individuals with dermatomyositis: corticosteroids, immunosuppressive agents, intravenous immune globulin (IVIG). Typically, corticosteroids are considered a first-line treatment,…
Prior to joining the Marine Corps, Jane Shepard was, for all intents and purposes, fairly healthy. However, after becoming stationed at Camp Lejeune in 1984, Jane’s health spiraled. She often…
Scientists affiliated with Massachusetts General Hospital (MGH) and Children's Hospital Philadelphia recently identified a novel mitochondrial disorder in a pair of identical twins. Doctors began studying these twins when…
According to a story from mirror.co.uk, Emma Timofte, age six, was diagnosed with a rare disease called incontinentia pigmenti. The diagnosis came when she was only a few weeks old.…
In 2005, The Chronicles of Narnia: The Lion, the Witch, and the Wardrobe brought us into the fantastical world of Narnia. Much like in the novels, the film's stars journeyed to…
Because it is Thanksgiving, the Patient Worthy team asked me to share my thoughts on gratitude. Some among us might think it would be a daunting assignment. After all, I have a…
People within the rare disease community face many barriers in regard to the diagnostic process. It can take years to receive an accurate diagnosis. Paired with medical costs and…
There is a certain time of year in which the trees change color, the air becomes crisp and we pour gravy on everything. As I am typing this, I am…
I love Thanksgiving! It's a great time to catch up with friends and family. The one drawback is my diet is strictly controlled due to my rare disease, so at…
Proton pump inhibitors (PPIs) are a class of medicine that reduce the amount of stomach acid production. They are commonly known for their use in acid-related disorders, such as gastroesophageal…
I love quotes. Some of them I read or hear and they go in one ear and out the other. Those that really resonate stick with me and help change…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
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