Two companies, GtreeBNT and YuYang DNU, will collaborate in a joint venture to develop the investigational drug RGN-137 for the treatment of epidermolysis bullosa. For more details, you can read…
The Boston-based biopharmaceutical company Berg LLC has announced that their experimental drug candidate BPM 31510 (ubidecarenone) has been awarded Orphan Drug designation by the US Food and Drug Administration (FDA)…
The National Center for Advancing Translational Sciences (NCATS) supports rare disease patients and their communities. They provide translational research funding, tools and other resources that are helping to address unique…
While she's constantly smiling and full of energy, little girl Makenzie Cadmus struggles with a rare condition known as epidermolysis bullosa, reports News 12 Long Island. The rare disease has…
A 9-year-old boy, whose family has decided to remain anonymous, is finding relief after suffering from an incurable skin disease. According to his father, the boy was near death and…
Epidermolysis bullosa (EB) is a rare disorder, in which a genetic mutation disrupts the production of a protein that holds the skin together. This creates intense and painful blistering externally,…
Last week, science continued to break boundaries and save lives. In this case, a new gene therapy was used by doctors in Europe to replace the decaying skin of a…
Two years ago, a seven-year-old boy from Syria was in dire condition. 80% of his skin was gone; his entire body covered in wounds and abrasions. He still had some…
Let’s talk clinical trials. Clinical trials are part of clinical research and at the heart of all medical advances. Clinical trials look at new ways to prevent, detect, or treat…
A health crisis—just like epidermolysis bullosa (EB)—is different for everyone. But, most have one thing in common: Bad news blindsides us. We take a hit—a hard one at that. When…
It’s always heartening to see scientists take up research in therapeutic areas that are under served and poorly understood. And when their research yields positive results, it’s downright amazing. Honestly,…
Maybe we should chart our own course in life like one sweet baby girl who was born with a rare skin disease called Epidermolysis Bullosa. Little Joella lives with the…
Who is excited for another long weekend coming up this Monday?! To kick off your three-day weekend, take a look at these intriguing stories... Researchers have identified a superbug that…
When we need it most, stories can inspire, encourage, and quite frankly, make us feel good. Especially if the story is about a super cute little eight-year-old girl who was…
Epidermolysis bullosa (EB) is a disorder characterized by blistering of the skin. It also affects all the body's mucous membranes, so while the blisters may be visible, the person's lips, tongue, mouth,…
Life is one wild ride, isn’t it? The greatest surprises of life may be just around the corner. My life was changed by one little red radio flyer wagon. An…
I always have a knee-jerk reaction that borders on anger when I read about babies who are dealt an unfair hand in the rare disease world. It seems incredibly unfair…
They are called, "butterfly children." These butterfly children earned their names for their fragile skin---said to be as delicate as the wings of a butterfly. The official name of…
Here's a riddle to start your day: What do acromegaly, Cushing's disease, and epidermolysis bullosa have in common? If you guessed that they all have treatments owned by a small pharma…
Epidermolysis Bullosa, or EB, is a connective tissue disorder that approximately 200 babies are born with each year. The most serious symptom is the extreme fragility of skin. People with EB suffer…
About two months ago, I bucked up and started recycling. (No this is NOT clickbait.) Now let me first explain that for years as a “responsible adult,” I did my…
Pictured: Dr. Jakub Tolar EB can make a lot of daily activities difficult and painful. Check out some tips and tricks compiled from presentations at the 2016 Debra CARE Conference.…
It’s always awesome to announce a new clinical trial, especially for something that could offer relief to people suffering from the rarest of the rare conditions. And rare doesn’t begin…
Can I just tell you how I tired I am of all the millennial hate out there? People are beyond harsh to the younger generations, and this article on Heritage High…
Imagine being 16 years old, and instead of meeting cute girls, going out with friends, and going to parties that get busted, you have to manage a rare, chronic condition…
© Copyright 2024 Patient Worthy
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.
