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Giving hope to thousands of families | Cold Spring Harbor Laboratory

It’s a parent’s worst nightmare, finding out your newborn child may never walk or even stand on their own. That used to be the reality for as many as one in 6,000 babies born with spinal muscular atrophy (SMA), a genetic disorder that causes extreme nerve damage, muscle weakness, and sometimes, paralysis. For a long time, doctors had to tell parents that children born with the most severe form of the disease likely wouldn’t live past age 2. Toda…

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Cold Spring Harbor Laboratory broke the news in on Tuesday, August 5, 2025.
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