Pregnancy hormone has unprecedented, powerful effect on spinal muscular atrophy

Although spinal muscular atrophy (SMA) is caused by the loss of a specific gene, all infants and children with SMA have an untouched highly similar gene within their genetic make up. Activation of this copy gene has the potential to treat SMA, and now researchers report the strongest such activation yet observed with attendant benefit on mice genetically engineered to have SMA.

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