FDA approves first gene therapy for inherited deafness, shown to restore hearing for children with rare condition

The US Food and Drug Administration on Thursday approved the first gene therapy for inherited hearing loss, a one-time ...
STAT

I’m a rare disease mom, and I finally have new hope for my son’s future

Stecker is a rare disease mother and advocate and served as assistant secretary of public affairs and deputy chief of staff at the Department of Health and Human Services. Every parent dreams of a ...
YourTango

Research Suggests Kids Raised In The 60s And 70s Developed 5 Rare Traits Many Kids Today Are Tragically Missing

Research suggests that kids who grew up in the 60s and 70s developed five specific traits that feel noticeably rarer in kids ...

Teen with Rare Cancer Didn’t Want to Throw Away Surprise Make-A-Wish Gift, Then He Had Idea for Kids Like Him (Exclusive)

Duncan Simmons, 17, underwent six months of treatment after being diagnosed with a rare, aggressive form of cancer ...
AOL

This rare disease destroys a child's ability to walk and speak. Could a new drug help?

Ever since her 15-year-old son Cole was diagnosed with a rare, progressive illness called Hunter syndrome when he was 2, Kim Stephens has been waiting. Waiting to see if Cole will lose his ability to ...