Girl with Kabuki Syndrome Has Waited 3 Years for Knee Surgery
When the COVID-19 pandemic swept the world, nobody knew what the short- or long-term effects would be. Now, a few years into the pandemic, we’ve begun understanding some of…
When the COVID-19 pandemic swept the world, nobody knew what the short- or long-term effects would be. Now, a few years into the pandemic, we’ve begun understanding some of…
There are a number of different forms of congenital hyperinsulinism, along with a variety of different causes: a mother’s unmanaged diabetes, premature birth, gene mutations. But up to half…
According to a story from ca.sports.yahoo.com, the biotechnology company Galecto, Inc. has announced encouraging findings from an intermediate assessment of its phase 2a clinical trial. This trial is evaluating the…
Prior studies have found a correlation between inflammatory bowel disease (IBD) and colorectal cancer; those with IBD are at an increased risk of developing this cancer compared to the…
A team of researchers at Shanghai’s Fudan University, acting upon the knowledge that ketogenic diets can boost liver ketone production, have recently examined the possibility of preventing thrombocytopenia (low…
What if this Christmas season, God would appear to me and say, “Ask what I shall give thee,” as He did in a dream to King Solomon many years ago.…
According to a story from empr.com, the Oncologic Drugs Advisory Committee, part of the US Food and Drug Administration (FDA), recently voted 16 to 0 against the investigational drug omburtamab…
In May 2016, I was diagnosed with a rare genetic neurological disease called adrenoleukodystrophy (ALD). Sadly, it's incurable and there's no medication to ease my condition. Since then, my life…
When Jordan Lambropoulos was younger, she seemed to always be ill. She struggles to remember a time in her childhood where she would characterize herself as “healthy.” Even doctors…
Do you wish to be heard? Recently PR Newswire announced that on the 28th of February 2023, NORD and the worldwide community of rare disorders will be raising awareness…
To read part 1 of Nikole's Holiday Confession, click here. I feel guilty when my limitations force others to have to alter their holiday expectations. Like, how do I tell…
Contrary to simply stopping tumor growth, the biomedical engineers at Duke University have successfully demonstrated a new treatment for pancreatic cancer in a mouse model that may make a…
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…
Pumpkin pie, ginger-bread cookies, candy canes, and chocolates. Oh what a sweet season! A few nights ago my kids and I spent a few hours crouched over the wafting smells…
According to a story from PR Newswire, the biopharmaceutical company AMO Pharma Limited has recently announced the completion of enrollment for its REACH-CDM clinical trial. This trial will be investigating…
Written by Lisa Ann Krutzik An inexpensive and extremely valuable gift you can give your older teen or adult child this holiday season... A letter from you. I spend a…
In a mid-November 2022 news release from biopharmaceutical company Apexigen, Inc., the company shared that positive interim results were available from a Phase 2 study evaluating sotigalimab, in conjunction…
We often hear people talking about the stress they are feeling during the holidays, while those in the rare disease community silently think, "You have no idea!" During this season,…
Med City News published an article by Bruce Feinberg, M.D. at Cardinal Heath, examining CAR-T treatments and the impact they have had in medicine during the past five years. CAR-T's…
For those of us who suffer from depression, that mostly invisible mental illness that, at its worst, can render us incapable of getting out of bed and, at its best,…
A new awareness campaign called RKD & Me has recently kicked off with the goal of spreading awareness of rare kidney disease (RKD). The project is a collaboration between the…
There are a number of risk factors associated with the development of inflammatory bowel disease (IBD). These include being younger in age, being Caucasian or of Ashkenazi Jewish descent,…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
According to a story from PR Newswire, the cell therapy company Cellevolve Bio, Inc., recently announced that the US Food and Drug Administration (FDA) has advanced the development of its…
Chronic pain reminds me of a bully that picks on kids in the schoolyard every day. Like the bully, chronic pain does not follow any rules and after multiple beatings,…