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Gene therapy aids boys with Lorenzo's Oil disease breakthrough
Gene therapy shows promise for adrenoleukodystrophy in a study of 17 boys ages 4 to 13. Fifteen remained neurologically stable two years after treatment, while two died from disease progression and transplant complications. Bluebird Bio funded the trial, with doctors weighing long term benefits and costs as regulatory review continues. Brandon Rojas, severely affected, now relies on a wheelchair and feeding tube, while his brother Brian benefited from early intervention.
MENTIONED IN THIS ARTICLE
David A. Williams
Boston Children’s Hospital
Paul
Liliana Rojas
Dover Plains
New York
Brian
Brandon
New England Journal of Medicine
Kansas City
Missouri
Bluebird Bio
Lorenzo
Marc Engelen
Emma Children’s Hospital
Amsterdam
U.S. Food and Drug Administration
Williams
Bluebird
U.S
Europe
Brandon Rojas
Katherine C. Cohen
Boston Children's Hospital
Child Neurology Society