Pancreatic cancer is rare, and often, patients don't show symptoms until they're already in the later stages. Top pancreatic cancer centers in the country have been working together to find…
Prader-Willi syndrome is a rare condition which causes the body to feel insatiable hunger, regardless of how much a patient eats. Treatment options are currently inadequate for many patients. Today,…
The Canadian Fabry Association wants to create awareness for rare diseases and you can help! They want to get as many people to wear a Be Rare Be You tattoo…
Caplacizumab is a medicine that has been developed to treat acquired thrombotic thrombocytopenic purpura (TTP). TTP causes serious symptoms. such as thrombocytopenia, a condition in which a patient doesn't have enough platelets. A…
Lennox-Gastaut syndrome (LGS) is a severe and rare form of epilepsy, which causes patients to often have multiple seizures a day. It generally appears in early childhood, and is difficult…
Hereditary hemochromatosis is a genetic form of a condition in which the body builds up too much iron. To read more about hemochromatosis, click here. La Jolla Pharmaceutical Company has…
Severe combined immunodeficinct (SCID) is a rough diagnosis, that leaves the immune system of a patient at high risk for serious infections. Orchard Therapeutics wants to develop a treatment SCID…
Happy Holidays, Patient Worthians! As we celebrate the holidays this year, we take a moment to reflect on the things that matter most deeply to us. This week, we have…
REGENXBIO Inc., a pharamaceutical company specializing in gene therapy, just shared that it launched a Investigational New Drug application to conduct Phase I/II trials on the new therapy they're developing,…
Pharmaceutical company and gene therapy leader, uniQure, published promising data from their current phase I/II trial testing out a gene therapy called AMT-060 in patients with serious cases of hemophilia B.…
If you've been waiting for a new rheumatoid arthritis treatment, good news is on the way! Rheumatoid arthritis is a chronic inflammatory condition that causes joint pain among other symptoms.…
The internet has been trying to figure out the caffeine question for years: is it good for us? Is it bad for us? Will it stunt our growth? Will it prevent…
Happy Holidays, Patient Worthians! As we celebrate the holidays this year, we take a moment to reflect on the things that matter most deeply to us. This week, we have…
There's a great need for more depictions of rare conditions in the media, and the film "Wonder" has put rare disease in the spotlight-- especially craniofacial conditions like Crouzon syndrome.…
All the odds were stacked against Jimmy Roberson, a man in Birmingham, Alabama. It all began in 2013. Jimmy was on a trip to the lake, but the relaxing day…
Chronic granulomatous disease (CGD) is a rare inherited primary immunodeficiency disorder that impairs the body’s ability to kill certain bacteria and fungi. As a result, people with CGD are at…
36-year-old Steve Pete has never felt physical pain in his life. While that may sound appealing, it comes with some serious downsides, such as literally chewing off his own tongue…
You may have seen Sanele Junior Xaba in an ad for Adidas Original, posing in mythical depictions for European artists, or photographed nude, covered by only butterflies in the magazine Label.…
Being a teenager is inherently difficult. There's no real way to make it to your twenties without feeling tired, cranky, and misunderstood by everyone around you. It's even harder when…
If you have a rare disease, chances are, sometimes you feel all alone in your experience. When you look around, there are people all over the world fighting similar battles.…
It was a classic story of boy-on-dialysis-waiting-for-a-kidney-transplant meets girl. The year was 1997. Larry Wetsch's kidneys were in dire state, hardening due to a rare disease called glomerulonephritis. Glomerulonephritis happens when…
Whenever I was looking for something I had lost as a kid, my mom would offer the same, unhelpful advice: It'll show up in the last place you look for…
Welcome Back Patient Worthians! This week, we have stories of brave patients and caregivers who run, march, and make innovative steps to reach recovery. A young girl marches after her…
Rett syndrome is a severe, genetic neurological disorder, which primarily affects girls. While it was once thought to be a form of autism, it's now understood as its own condition.…
Imagine waking up one morning feeling pain shoot all over your body. It seems like it's out of nowhere. When you try to move your neck, it will barely budge.…
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