Gene therapy helps boys with ‘Lorenzo’s Oil’ disease

In this Aug. 29, 2017 photo provided by Boston Children’s Hospital, Dr. David Williams, chief scientific officer at Boston Children’s Hospital, visits with the Rojas family from left, mother Liliana, son Brandon, father Paul and son Brian at Boston Children’s Hospital. The young boys suffer from adrenoleukodystrophy, an inherited nerve disease featured in the movie “Lorenzo’s Oil.” Brian was able to benefit from an experimental treatment while the disease had progressed too far in Brandon to qualify for the gene therapy study. “This appears to be a terrific new therapy” for many kids who had little hope before now, said Williams, a leader in the study. (Katherine C. Cohen/Boston Children’s Hospital via AP)


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