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 Saudi medical team was able to surgically remove 20 kilograms (44 pounds) of weight from a young girl with a rare disease. Five-year-old Safia, who suffers from Prader-Willi 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…
Birthday’s are important milestones for parents. First birthdays especially. For children with Costello syndrome, however, celebrating doesn’t always come naturally. After many difficulties, Joey and his mom finally shared that…
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…
A new Brazilian study indicates that Zika is hitting especially hard for lower-income groups, with infection rates exceeding 60%. Zika is, of course, the virus that spread rampantly a few…
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,…
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.…
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.…
Bradley Lowery won the hearts of many as a football mascot. After an ongoing battle with neuroblastoma, he lost his life earlier this year. Neuroblastoma is a rare form of…
Press Release: Lend Your Voice to the Fight to Breathe Faced by the UK’s Cystic Fibrosis Community Cystic fibrosis families from around the UK have joined forces to release a…
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…
When you suffer with severe atopic dermatitis you can get pretty desperate to do anything to relieve the itch and other symptoms. So taking a bleach-bath is not out of…
Brielle Manning has been fighting through seizures for over a year, but the dust has settled and her family is now seeing some relief. Brielle suffers from an incredibly rare…
Leukemia is a deadly serial killer that will claim more than 24,500 lives this year, but the Independent Citizens Oversight Committee of the California Institute for Regenerative Medicine (CIRM) just…
The Virginia Newborn Screening Advisory Committee met late last week to determine the fate of Pompe Disease and MPS-1 screening in VA. Before the vote took place, some families spoke…
A teen suffering from a rare disease is receiving some holiday love from kind strangers. When Nathan Kelley started losing his hearing, he knew something was wrong. At first, him…
When Raeno Brendtro was asked to sit down after her diagnosis, she refused. She needed to stand for as long as she could, now that her time was limited. The…
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…
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