Stories about: gene therapy

Cole: Having a ball after CAR T-cell therapy

Cole, who received CART T-cell therapy for ALL, poses with his twin brother before a baseball game.
Cole, left, and his brother, Michael [PHOTOS COURTESY OF MALONE FAMILY]

After undergoing a promising new treatment for acute lymphoblastic leukemia (ALL), Cole Malone is back to doing what he loves: playing on a flag football team with his twin brother, Michael.

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Manny: Hoping new research helps others with sickle cell disease

Manny, who is in a clinical trial for sickle cell disease, is examined by his doctor.
Manny at a visit with Dr. Esrick [PHOTO: SAM OGDEN/DANA FARBER]
Emmanuel “Manny” Johnson, Jr., shares many loves with his little brother, Aiden — from basketball to video games. One thing he wishes they did not share is sickle cell disease (SCD), so Manny is playing a role in a new effort to improve treatment for patients like 7-year-old Aiden, himself and others living with the inherited blood disorder.

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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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Gene therapy trial offers hope for Harry

Boy with Wiskott-Aldrich syndrome holds father's hand.
Harry and his father at Dana/Farber-Boston Children’s

In their Brookline home-away-from-home, 2-year-old Duy Anh “Harry” Le plays with blocks and pop-up toys on the floor with his mother, Thao Nguyen. He is lively and happy, and his skin is clear. He looks almost nothing like the sickly baby covered in eczema who arrived in Boston from his native Vietnam in November of 2016 to participate in a gene therapy clinical trial for Wiskott-Aldrich syndrome.

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