Gene therapy helps boys with "Lorenzo's Oil" disease
October 5, 2017
The fledgling field of gene therapy has scored another win: An experimental treatment seemed to help boys with the inherited nerve disease featured in the movie "Lorenzo's Oil."
Fifteen of the 17 boys treated in a study had no major disability two years later — remarkable for a disease that often causes swift decline and kills within a decade.
"This appears to be a terrific new therapy" for many kids who had little hope before, said one study leader, Dr. David A. Williams of Boston Childrens Hospital.
The news is bittersweet for Paul and Liliana Rojas, a couple from Dover Plains, New York...