The space between heartache and happiness: Two sons with adrenoleukodystrophy

The Rojas family at a recent visit to Boston Children’s. When Paul and Liliana Rojas talk about their life, they describe it in one of two ways — the way it was before their sons, 10-year-old Brandon and 7-year-old Brian, were diagnosed with ALD, and the way it is after. Their story is one of heartbreak — but also hope, in the form of a new clinical trial. Learn more about the results of the clinical trial, recently published in the New England Journal of Medicine, that halted the progression of Brian’s ALD. ALD is short for adrenoleukodystrophy, a debilitating brain disease that simply goes by its initials. “Life before ALD was pure happiness without worries,” Paul says. “It was anything a parent could wish for — two boys with no medical issues, active, athletic, the healthiest boys ever.” The two were inseparable. They played sports together in their hometown of Dover Plains, New York; idolized superheroes; danced like crazy; and dreamed of someday inventing video games. Brian was Brandon’s shadow. Brian and Brandon, “before ALD” The first signs of ALD It all began with a phone call from Brandon’s school in May of 2014. “They asked, ‘Is everything OK at home?’” explains Paul. Brandon, then a 6-year-old first-grader who excelled at reading and math, was showing signs of fatigue and an inability to focus. “Nothing is going on, everything is good. I am good,” Brandon told his mom and dad. “Like any parent, you let things play out,...
Source: Thrive, Children's Hospital Boston - Category: Pediatrics Authors: Tags: Diseases & Conditions Our Patients’ Stories Research and Innovation adrenoleukodystrophy ALD clinical trial Dana-Farber/Boston Children's Cancer and Blood Disorders Center David Williams MD gene therapy Lorenzo's Oil New England Jour Source Type: news