‘It’s not a cure’: A gene therapy is opening a new chapter for children, but challenges endure

WEST BOYLSTON, Mass. — When Rachael and Pat Brown rattle off all that their daughter Kate can do, their voices are imbued with an astonishment that eclipses even the usual parental pride. Never mind that the skills might seem meager for a 3½-year-old.

Kate can pick up toys. She can scooch herself along the floor. She’s strong enough to sit and hold her head up, and that means she can join them at the dinner table and needle her older siblings. She can talk, well enough that she can ask her parents to turn on Genesis’ “Invisible Touch.”

“And,” Rachael said, “she’s alive.”

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