Rare Disease in the News
Lebanon girl, 4, dies after sister succumbs to same rare disease
Doctors at Cincinnati Children's Hospital Medical Center were perplexed until 2014, when one doctor found a genetic test for infantile neuroaxonal dystrophy, an ultra-rare disorder in which iron floods the brain until it short-circuits. Life expectancy ...
'Karaoke Queen' sings through her battle with rare disease
"I was inspired by Taylor Swift," Audrey said, "because she has all the passion and spirit and I thought I would start singing to maybe put a little passion and spirit in me so maybe I could fight off my disease." Her disease is Diamond Blackfan Anemia.
7-Year-Old's Viral Videos Draw Attention to Rare Disease
Seven-year-old with rare disease becomes a singing Internet sensation
Kansas City Star
Finally diagnosed with rare disease, mom gets zip back
Kansas City Star
Primary immunodeficiency diseases make up a group of more than 250 rare, chronic disorders that involve the body's immune system being disabled or working improperly. This often results in recurrent infections of the skin, respiratory system and the ears.
Amid Tragic Loss, A Family Fights For Cure For Rare Disease
CBS 2's Brad Edwards has this Original Report. Noah VanHoutan died in March, not yet 12 years old, of Batten disease, a rare genetic degenerative neurological disorder. “It's a universally fatal disease, currently with no approved therapy or cure ...
Britain's first 'robo-dog' given bionic legs after rare disease ravages pooch's paws
Robbie the Shetland Sheepdog has been transformed into a robo-dog thanks to his new revolutionary new legs. The 11-year-old canine was plagued by problems in his hind legs before he was given his artificial feet. Owners had feared they would have to ...
'Spicerfest' shines spotlight on rare disease
Wayne, a DeSoto County artist, said he plans to also dedicate a portion of abstract art collection to help finding a cure for the disease that has almost ravaged Spicer's physical body but not her spirit. "When people put it on their wall, I hope it ...
Fundraiser Held for Auburn Teen Battling Rare Disease
Despite the disease racking Juliette's body, those around her say she has a lot of fight in her. She's been diagnosed with mixed cognitive tissue disease with poluarticular idiopathic arthritis. Some days are more difficult than others. “She has gone ...
UT Softball makes girl with rare disease part of the team
KNOXVILLE, Tenn. Call it fate, the right place at the right time, or an act of God. That's what happened with Tennessee Softball co-head coach Karen Weekly. She was at a Friday night high school game when it was put on a rain delay. Sitting in her car ...
Wall Street Journal
Senators push FDA to approve drug for rare disease
Ron Johnson, R-Wis., and Dan Coats, R-Ind., are disappointed that a panel of experts did not recommend a new drug called Sarepta for Duchenne muscular dystrophy, a fatal disease that mainly affects young boys. The senators' call follows a massive ...
FDA Takes More Time on Muscular Dystrophy Drug Approval
Is the FDA's decision to delay Sarepta drug decision good or bad? Nobody really knows
FDA delays ruling on Sarepta's muscular dystrophy drug