Spinal Muscular Atrophy

Zolgensma’s approval, and that of Spinraza, is the beginning of a new paradigm for treating SMA and for the fight against muscular dystrophy in general. Without any treatments, SMA would remain the top genetic killer of children under 2 - Nathan Yates

Spinal Muscular Atrophy

image by: Cure SMA
     

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

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.”

Kate has spinal muscular atrophy, a genetic disease that causes the progressive loss of muscle function and that…

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Last Updated : Saturday, November 13, 2021