A new study may lead to new cures and treatments for Marfan syndrome. Researchers at Johns Hopkins have made great progress testing in mice. Follow the original story here. On…
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!…
5-year-old Gia Danninger was diagnosed with aplastic anemia last year. Ever since, it's been a constant battle for her life, but Gia fights with courage. This rare blood disorder causes…
After dueling with a fatal rare disease, Makenna Vanzant, will be making her epic return to the basketball court. The high school sophomore has been battling hemolytic uremic syndrome and…
What do Nathaniel, a child with Bardet-Biedl Syndrome and Sylvester Stallone's famous boxing hero have in common? Keep reading below, or follow the original story to find out! Braille reading…
On this season of America's Got Talent, the world met Christian Guardino. I was one of millions who heard his beautiful singing voice as he belted out his song onstage.…
Patients diagnosed with Duchenne muscular dystrophy (DMD) have always lived with a grim prognosis. Many don't make it into their 20s and face debilitating symptoms years earlier-- a dark reminder…
Jay and Amy Granzow are among the many worried parents fearing the GOP tax plan, because of the threat it brings to orphan drug research. The Manhattan Beach couple fear…
Robbie Edwards has a rare disease, one so rare that she's one of only 17 people in the world with it. She suffers from hereditary spastic paraplegia which represents a…
As a scientist, Mary-Claire King has had a remarkable career related to her study of BRCA genes. She’s received a Lasker Award and has been recognized by President Obama with…
Not all bacteria are created equal, but all play a role in sustaining--or breaking down--health. And when you're talking about children with cystic fibrosis (CF), a lung disease that impairs…
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…
In the UK, Newcastle University and Pfizer are collaborating on a $39.9 million-dollar funded project aimed at developing greater tools for testing patients at the most risk for non-alcoholic fatty…
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…
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.…
A small town Australian mayor was caught parking in a designated handicapped spot, and the mother of a rare disease girl is not having it. The event took place at…
A video of former US Vice President Joe Biden on the talk show The View comforting co-host Meghan McCain has been making the rounds on the Internet the last few…
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…
The FDA has granted a special designation for a drug company developing a gene therapy for the treatment of leber congenital amaurosis (LCA). MeiraGTx received rare pediatric disease designation for its…
During a time when we get a lot of bad or polarizing news about the world, it's important to highlight the good stuff - so here is some good stuff…
Because of an extremely rare disease, 3-year-old Reshmi's head has doubled its size after a fluid buildup in her brain. Reshmi Marak lives in a poor village in Tripura, north…
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…
Back in 2014, the FDA gave the green light for eight brand new drugs for orphan diseases. Among the pack was BioMarin Pharmaceutical's very own VimiZim. Weird name right? Well,…
A simple facebook post has led to a life changing event for a young boy with CLOVES Syndrome. Keep reading to find out how, or follow the original story here.…
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