We all know that an early diagnosis can save lives. But, in an unexpected turn, this mother’s late diagnosis is what saved her son. RareConnect.org details the story of Malinda…
A new gene therapy treatment may be on the horizon for Oculopharyngeal Muscular Dystrophy patients. Find out more below, or keep up with the original story at musculardystrophynews.com. Oculopharyngeal Muscular…
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…
A much-lauded piece of wisdom in engineering is that “failures teach more than successes.” While this might seem to contradict itself, I ask you to think back to your own…
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.…
If there was a proven medication to slow the progress of a chronic and deadly lung disease with no cure, why wouldn't you take it? That's the question that researchers…
For people suffering from severe atopic dermatitis (AD), commonly referred to as severe eczema, relief may be in sight, according to a recent article in Dermatology Times. If you're one of…
University of California, San Francisco scientists have developed a new vaccine that treats diffuse intrinsic pontine glioma (DIPG), a rare childhood brain tumor. To learn more about DIPG, click here. Great…
I almost died at birth and had to wear metal leg braces and shoes for the first two years of my life, an attempt to straighten my inward facing rigid…
Each year pharmaceutical companies spend millions developing and testing potential treatments. These drugs have to go through many levels of testing to make sure they are safe and effective. And…
The close knit Algiers community in New Orleans united together once again for a charity walk to raise awareness and money for Stevens-Johnson Syndrome (SJS). SJS is a highly rare…
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…
4-year-old Dimi loves to hang out with other preschoolers his age, play in the park, and ride his bike like a regular kid, but his mother Frances is fearing his…
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…
Great news for the rare disease community! Ultragenyx Pharmaceuticals reported back positive results from its phase 3 study of the treatment drug burosumab in adults with X-linked hypophosphatemia (XLH). XLH…
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…
Our gut bacteria may be responsible for antiphospholipid syndrome (APS). What do you need to know about the ordinary gut bacteria known as commensals, and APS? Keep reading, or view…
The highly anticipated Season 2 of "The Crown" hit Netflix today and with it, a new chapter of political intrigue, elegant royalty and... a drug addicted JFK? Say what? It's…
Tayla Udall from Essex, England, is used to finding her four-year-old son rummaging through leftovers in the kitchen because of his rare disease of perpetual hunger. Frankie suffers from Prader-Willi…
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.…
Lilia Zaharieva was a University of Victoria student in her early late twenties when she found herself losing half her leg function due to cystic fibrosis. Walking from class to…
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.…
Canavan disease is an isolating condition. It's a rare disorder caused by a genetic mutation that can be passed on in families. It damages nerve cells, so they're not able…
Amir Khan is one of the leading athletes from Singapore. He competes in mixed martial arts, and has made a name for himself in ONE Championship. He's tough, fast, and…
African Americans face a far higher danger of developing multiple myeloma-- especially African American men, who run three times the risk of a diagnosis. These groups are also about half…
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