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…
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