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Early treatment is a lifeline for infants with SMA - Nature Medicine

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Early treatment is a lifeline for infants with SMA - Nature Medicine

In the phase 3 SPR1NT trial, pre-symptomatic gene therapy demonstrated impressive clinical outcomes in infants with a genetic diagnosis of spinal muscular atrophy (SMA); long-term safety follow-up of these patients must now be a key priority.

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Early treatment is a lifeline for infants with SMA - Nature Medicine

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Early treatment is a lifeline for infants with SMA - Nature Medicine

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