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.
The Phase 3 PLEO-CMT clinical trial evaluated the safety, efficacy, and tolerability of PXT3003 for patients with Charcot-Marie-Tooth disease type 1A (CMT1A). Altogether, 323 people enrolled. During the trial, researchers…
Ryan Smith has always been tenacious in pursuing his goals. One of his most important goals? Supporting his family and his community. As a lieutenant with the Stillwater Fire Department,…
There are a number of different mosquito-borne illnesses. But sometimes it can be difficult to make an accurate diagnosis when these illnesses – and many others – share similar symptoms…
How do certain conditions contribute to mental health, depression, and anxiety? It's no secret that having a rare, chronic, or underserved condition can be isolating. But what are the actual…
When Brett Devloo was fifteen years old, he lost his sight. It happened, he feels, suddenly; one minute, Brett was reading and writing notes in his history class and…
Human papillomavirus (HPV) types 16 and 18 play a huge role in the development of cervical cancer. In fact, an estimated 70% of cervical cancer cases result from these…
According to an August 2022 news release from biotechnology company Elixiron Immunotherapeutics (“Elixiron”), EI-1071, a therapy for patients with idiopathic pulmonary fibrosis (IPF), recently earned Orphan Drug designation from…
Currently, there are an estimated 30 million people within the European Union (EU) who have a rare disease. The “Orphan Medicines” (or Orphan Drug) program was designed to facilitate…
September is a wonderful month to raise awareness. So mark down September 24th on your calendars for the 8th international Atypical Hemolytic-Uremic Syndrome (aHUS) Awareness Day! aHUS Awareness Day is…
Effective treatment options are crucial for patients with Chagas disease who show serious or life-threatening symptoms. According to Infectious Disease Advisor, a Phase 2 study in its proof-of-concept phase evaluated…
Many patients with ankylosing spondylitis (AS) could be treated with biologics – in particular, biologic disease-modifying antirheumatic drugs (DMARDs). However, this line of treatment is not efficacious nor well-tolerated…
Missense mutations cause Angelman syndrome in around 5% of diagnoses. However, researchers have often been unclear or unsure of the biochemical consequences of these missense mutations on the affected…
On September 9, 2022, researchers presented data at the European Neuroendocrine Association (ENEA) meeting. According to Yahoo! Finance, one such presentation centered around new safety and efficacy data from the…
Have you ever heard of Orphan Drug designation? This designation is intended to spur research and development into drugs or biologics for rare diseases. Orphan Drug designation is granted…
For many people with Chagas disease, treatment may not be necessary; they stay asymptomatic and don’t experience many, if any, health effects. Some may experience mild flu-like symptoms. Typically, doctors…
Together, Chris Lucas and Preston Brust make up LOCASH (also written as LoCash), an American country music duo. While the pair puts out some absolutely amazing songs (trust me), Preston…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare conditions. A "rare" condition is defined as one affecting fewer…
For five years, 20-year-old Christopher Backlund lived with his grandfather, with whom he had a strong and meaningful bond. Unfortunately, Christopher's grandfather, Mitchell, passed away in Februay 2021. As Christopher…
Victoria Acosta has always been an active child. The bubbly two-year-old, who currently lives in Tennessee, is described by her mother Andrea Rocha as constantly moving. However, it can be…
In a late July 2022 news release, immunodermatology innovator KSL Beutner Laboratories shared that it had launched an indirect immunofluorescence (IIF) serum blood test within the United States. The IIF…
Within the first few months after Reuben Jayce-Mills was born, he seemed to be progressing fairly normally. But when Reuben turned nine months old, his parents noticed some concerning…
Every day, Jackson Garwood’s parents, Darren and Rebecca, look at him and smile. There were points when they didn’t expect to have their son for this long. You see, Jackson…
Both Dravet syndrome and Lennox-Gastaut syndrome (LGS) are characterized by frequent and often severe seizures. In many cases, those affected require multiple medications to manage their condition and prevent epileptic…
Unfortunately, the drug development process can be difficult, especially in the rare disease sphere. Many drug developers choose to focus on larger disease states, as these are typically more…
Actor Max Adler is known for a variety of roles in films and TV shows such as Glee, Saugatuck Cures, and Switched at Birth. But did you know that…
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