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…
The 1st International Workshop on Myelodysplastic Syndromes was held this year in Miami, FL from June 24-26. This in-person event was a convergence of researchers and clinicians to discuss advances…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Although kinase-targeting drugs have been used for many years in the fight against AML and other cancers, according to a recent article published in FierceBiotech, scientists at the Cold Spring…
Medical science takes a giant leap with a process that sees tumors disappearing or significantly shrinking in 63.3 percent of cutaneous squamous cell carcinoma (CSCC) patients. According to a recent…
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…
According to a story from Yahoo News UK, Dr. Paula Hunt had a CT scan earlier this year with alarming results. The scan appeared to show a large tumor in…
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…
Written by Dennis Lodge Inclusion in the workplace is a big thing for virtually every major corporation in the world, however, the World Economic Forum statistics say that merely 4…
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…
A recent article in SciTechDaily explains that the many failures of newly created Alzheimer’s drugs are the result of clinical plaque tests that do not identify the disease in its…
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…
Mia Maysack is a Pain News Network columnist as well as the founder of Peace & Love life coaching practice for the chronically ill. Mia shares her ongoing saga with…
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…
According to a story from klfy.com, 15 year old Mia Braseaux may seem like a normal teenager; she plays sports, goes to school, and socializes with friends. However, Mia was…
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…
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