Without a risky bone-marrow transplant before symptoms appear, children with brain disease ALD can expect to live no longer than five years as nerve cells in the brain die off and erase one's ability to walk, talk, and think.
Even a successful transplant can result in permanent disabilities, reports the New York Times. That's why a new study in the New England Journal of Medicine is raising eyebrows: For the first time, doctors say they have successfully repelled adrenoleukodystrophy using gene therapy, a feat that could boost the survival rate of the rare brain disease affecting one in 20,000 boys, usually around age 7.
Two years after gene therapy—a treatment expected to cost hundreds of thousands of dollars—15 of 17 boys with a mutated gene causing ALD showed no symptoms. Of the two who died, one had ALD too advanced to be halted, while the other withdrew from the study.
- Laundry Workers Made Awful Discovery. Now, a Lawsuit
- FDA Says Bakery's Granola Can't Be Made With 'Love'
- 2 States Sue Over Trump Decision on Birth Control
Photo: Getty Images