A RARE AND FATAL DEGENERATIVE DISEASE THAT CAUSES PROGRESSIVE DAMAGE TO NERVE CELLS AND LOSS OF WHITE MATTER IN THE BRAIN. GETTING THAT DIAGNOSIS WHEN FAMILIES WOULD GET THAT, THE DOCTORS WOULD GIVE ...

Rithvik wasn’t dealt an easy hand in life. His first symptom — a distended abdomen — was identified during his birth ...

A new approach... Lucy Landman was born with a very rare genetic disorder that causes severe intellectual disability, weak muscles and seizures, among other symptoms. "She is expected to very much ...

Lucy Landman was born with a very rare genetic disorder that causes severe ... to change that by raising money to research single-gene disorders like her daughter's. One day, while out fundraising ...

Lucy Landman was born with a very rare ... from one disease to another and not take four years and $10 million to do that,“ Urnov says. The approach from one patient to the next would be essentially ...