Growing up, Matthew Gee always seemed to love football. He played as a linebacker for the 1989 Trojans at the University of Southern California and was part of the 1990…
Each day, an estimated five children within the U.S. are born with congenital diaphragmatic hernia (CDH), a diaphragmatic defect which is fatal in approximately 33% of cases. While these hernias…
It’s no secret that more mathematics, data, and technology are being used to drive advances within healthcare and medicine. For example, we’ve seen the heightened use of artificial intelligence (AI)…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
What is the underlying cause of cancers such as esophageal adenocarcinoma or intestinal gastric cancer? Are there certain precursor cells that these cancers arise from? According to a news release…
More rare conditions are discovered as we learn more about the human body and genetics. For example, shares an article in Medical XPress, a research team from the University of…
Clinical trials play a crucial role in better understanding rare diseases and therapeutic interventions. According to a news release from rare disease company Albireo Pharma, Inc. (“Albireo”), enrollment is now…
Verge Genomics, based in San Francisco is one of a series of biotechs that have entered into a new era using AI in drug discovery. The company is backed by…
Oxytocin has been deemed the "love drug" or "love hormone." This hormone, which controls aspects of behavior and reproduction, is often released when we're in love or in physical…
According to a late October 2022 news release from gene therapy company AVROBIO, the sixth patient has been dosed in a Phase 1/2 clinical study evaluating AVR-RD-04 for patients…
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…
Before you read on, don't forget to check out Part 1 of our interview with Jennifer Wallace Valdes, PT. In Part 1, Jennifer discusses her background, what Duchenne muscular dystrophy (DMD) is,…
The European Society for Gene and Cell Therapy (ESGCT) recently held its Annual Congress from October 11-14, 2022. During the ESGCT Congress, various stakeholders discussed data, trends, and new research…
The Orphan Drug Act was passed in 1983 to stimulate the development of therapeutics for rare diseases. With this came the Orphan Drug designation. This designation is granted to drugs…
Glioblastoma multiforme (GBM) is notoriously difficult-to-treat. This cancer is both aggressive and invasive, spreading “tentacles” and becoming hard to remove even with surgery. As a result, finding more effective therapeutic…
The FDA created the Rare Pediatric Disease Designation and Priority Review Voucher Program to stimulate the development of therapies intended to treat, diagnose, or prevent rare pediatric diseases. These are…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose a rare disease or condition. Rare conditions are those affecting fewer…
According to a story from prnewswire.com, the pharma company Harmony Biosciences Holdings, Inc., has recently announced the release of data from its phase 2 clinical trial. This trial is evaluating…
According to ABC News, cases of Vibrio vulnificus (V. vulnificus) infections have risen in Florida following the devastating effects of Hurricane Ian. V. vulnificus infection, which may also be referred to as vibriosis, is…
Occupational therapy for individuals with Duchenne muscular dystrophy (DMD) requires a unique and specialized approach to maximize benefit and reduce harm. It is incredibly important that those performing occupational therapy…
Did you know that November is National Family Caregivers Month? Celebrated annually, this month is designed to recognize the contributions of caregivers and honor their actions. In a recent…
At 29 years old, Morgan Nuchols is living the fullest life that she can. She loves challenging herself by doing puzzles. But her favorite activity? Making birthday and holiday cards…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A rare condition is one affecting fewer…
Glioblastoma is notoriously difficult to treat and manage. The cancer is not only aggressive and quickly spreading, but is often treatment-averse. Because of this, glioblastoma comes with a poor prognosis…
For the last few decades, there has been increased interest in understanding the effects of sleep (or lack of!) on our overall health. A recent study sought to understand the…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
