News tagged with enzyme replacement therapy

Cancer drugs help the hardest cases of Pompe disease

Kids with Pompe disease fail because of a missing enzyme, GAA, that leads to dangerous sugar build-up, which affects muscles and movement. An enzyme replacement treatment pioneered at Duke University has saved many lives, ...

Jan 05, 2012
popularity0 comments 0

"Saving Eliza" campaign helps another child

Valerie Byers had long suspected that her son Will's diagnosis of autism was wrong. So when she saw a clip on the homepage of the Today Show about a little girl named Eliza, in late February, she knew instantly that 5-year-old ...

Jul 17, 2015
popularity27 comments 0

Subscribe to rss feed