Stories about: ALD

The space between heartache and happiness: Two sons with adrenoleukodystrophy

Paul and Liliana Rojas with their sons, Brandon and Brian, both of whom have adrenoleukodystrophy (ALD).
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.

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Forty years waiting for a cure: ALD gene therapy trial shows early promise

adrenoleukodystrophy-Ethan-Emily
A small piece of notepaper, folded twice, sits tucked in a slot of the secretary desk in the living room. Every so often, I pull it out, read it, then reread.

Addressed to my mom, the paper has a question and two boxes, one “yes” and one “no,” written with the careful precision of a 7-year-old.

I am sad of Ethan. You too?

A check marks the box.

— Yes. Yes, I am sad too.

Read Ethan’s story on our Vector blog and learn more about the ALD gene therapy trial.

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