The new nonprofit Every Cure has been created on the basis that every drug currently on the market effects multiple areas in the body. This premise has been proven…
Before you continue, make sure you've read Part 1 of our interview, where we discussed the Lord family's story and experience with rare disease, and the development of MobiMedQR. This tool, which…
Jack Johnson was diagnosed with Fabry disease when he was just seven years old. He likes to compare living with the rare disease to long COVID, but unlike long COVID,…
According to a story from the Glasgow Times, David Steed of Paisley, UK, was diagnosed with adrenoleukodystrophy when he was 18 years old. At the time, he didn't have any…
An article in BioSpace announced that the FDA’s CNS committee met twice in recent months to discuss the merits of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). Within…
October 5 is recognized each year as Progressive Familial Intrahepatic Cholestasis (PFIC) Awareness Day, a time to help spread awareness about this rare disease among the medical field and the…
If you haven't already, don't forget to go and read Part 1 of our interview. In Part 1, David Ogman and I discuss his son Jordan's TECPR2 diagnosis, the diagnostic journey,…
When asked his most important piece of advice for families within the rare disease community, Jeffrey Lord said: Do what you can control and move on from there. Every day,…
Imagine feeling like you have a sweat bee stuck in your eye socket that randomly stings you. The sting hurts for seconds. My left eye is red and tearing. My…
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…
In his early stages of life, Austin (A.J.) Rose was diagnosed with an ultra-rare disease called pontocerebellar hypoplasia type 6 (PCH6). Now, at three years old, A.J. is a…
There seems to be no limits when it comes to what a parent will do for their child. And when it came down to it, David and Stacey Ogman knew…
In late September 2022, biotech company Oncoheroes Biosciences, Inc. ("Oncoheroes") shared via news release that its product dovitinib, which the company has an exclusive pediatric licensing agreement for from…
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…
According to a story from Yahoo! News, a mother that is well attuned to her children can often have a special intuition when something is wrong. Thirteen year old Sheldon…
This was the fifth time Yadi Martin was pregnant. The first four pregnancies ended in miscarriages. But this time it was different. The fetus had a heartbeat. As Yadi and…
Over the last few years, there have been slight increases in the number of acute flaccid myelitis (AFM) cases throughout the United States. Specifically, 2014, 2016, and 2018 all saw…
In a late September 2022 news release from AVROBIO, Inc., the company’s gene therapy candidate AVR-RD-04 recently earned Rare Pediatric Disease designation. This investigational therapy is being developed as a…
Neuroscience News recently published an intriguing article suggesting that letrozole, a drug developed to treat breast cancer, has the potential to treat brain tumors. The discovery began with a question…
According to an article in Batten Disease News, enrollment is complete for a Phase 1/2 clinical trial. Within the trial, researchers will be evaluating Batten-1 for adolescents and adults with…
Osteosarcoma is genomically complex, which means that – sometimes – it can also be difficult to treat. Treatment and management, however, are crucial to ensuring positive patient outcomes. Doctors and…
Happy Friday! A tropical storm system will be traveling up through the mid-Atlantic this weekend, bringing wind and rain. Hunker down! This week, we have stories on Duke University being…
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…
Seven-year-old Rydder Ames of Hamilton, Ohio began having troubling symptoms five months ago. His mother, Krista Steven took him to the ER after a week-long headache followed by a…
According to a press release, the gene therapy company bluebird bio, inc. has recently announced that its gene therapy elivaldogene autotemcel (marketed as SKYSONA) has received Accelerated Approval status from…
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