Over the years, there have been growing concerns about medical research failing to cover or explore diverse groups of people. But in a world where healthcare is not just one-size-fits-all, how…
Growing up, Will Kleemeier learned all about polycystic kidney disease (PKD). He had several family members with PKD, so he knew that there was a chance that he too…
It’s October, so you know what that means – it’s Homocystinuria (HCU) Awareness Month! The goal of HCU Awareness Month is to raise awareness of homocystinuria throughout the medical…
Patient Worthy is proud to announce that we have recently partnered with Life on the Level, a support group and advocacy organization based in Leicestershire, United Kingdom. Life on the…
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…
From October 3-21, 2022, HAE Junior, a Patient Worthy partner organization, will have its art exhibit on display in the halls of the Czech Parliament. HAE Junior is an advocacy…
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…
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…
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