Irish drugmaker, Shire, announced earlier this month it will bring new genetic disorder drugs to India, stepping up its worldwide presence. Shire is unique in that it's one of the…
Pharmaceutical company, Pfizer Inc., announced recently that the U.S. FDA approved the first (and only) Janus kinase inhibitor for treating severe psoriatic arthritis (PsA). Called XELJANZ® and XELJANZ® XR, this…
Originally reported by Fox 17 Nashville, many Tennessee residents stress that a new tax reform bill will eliminate continued drug advancement and development for many rare diseases. The House and…
Kei Otake is a 19-year-old cello student at The Juilliard School - a prestigious performing arts college in New York City that boasts alumni like Viola Davis, Robin Williams, Adam Driver…
Gene therapy has been on the horizon for years now, but before today, it was only a reality for patients with non-inherited diseases caused by genetic mutations. That just changed!…
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…
Although Parkinson's disease (PD) affects men and women equally, researchers have discovered an area of disparity: care giving. A study published this month in Neurology by researchers at the Perelman School of Medicine…
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…
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.…
Global Genes is one of the world's most prominent rare disease advocacy groups and early next year, they will host the second annual RARE in the SQUARE event. At this…
Six weeks after their daughter was born, a young New Zealand couple had their two children taken away by the state (one was a newborn baby) because of what state…
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…
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…
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…
In Tecoman, Colima, western Mexico, Luis Manuel is now a ten month old baby, but he weighs as much as a nine-year-old. Luis Manuel tops the scales at 66 pounds,…
Fourteen years ago, Sabrina "Bria" Baumann's mother, Sophia, saw a mass growing in a sonogram. Something was wrong. Whatever was growing was on her chest and her neck, and the…
In celebration of her final 18th round of chemo, a brave 12-year-old girl was given a guard of honor march along the halls of the hospital. Her name is Alice…
Sandra De Santos has thousands of bubble-like tumors on her body due to a rare disease called neurofibromatosis type-1. It's a hard life, but she has spoken out proudly about…
This isn't the first time Barry Dubois, host of the Australian cooking show, Living Room, has had cancer. Seven years ago, he was diagnosed with plasmacytoma myeloma. It was different the first…
I want to be Isaiah Acosta when I grow up. The 17 year-old from Phoenix, Arizona was born without a jaw, due to the very rare disease situs inversus, which…
Happy Friday Patient Worthians! Gotta love that hectic-catch-up-crazy feeling post-Thanksgiving and pre-December holidays! We have heartwarming story of a little girl getting the ultimate gift for the holidays. We also…
Some people with Marfan syndrome look the part. They may be unusually flexible, with long arms, legs, and fingers, and a tall, willowy build. Some patients have scoliosis, crowded teeth,…
Glenda Thompson is a proud grandmother who will stop at nothing to ensure her grandson lives a comfortable life. 4-year-old Cole Thompson is suffering from a rare genetic disorder called…
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