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(Ivanhoe Newswire) - Imagine your baby ... spinal muscular atrophy,” explained Dr. Tilton. Now, the first FDA-approved oral ...
A prenatal test had shown that her fetus held two genetic mutations indicating type 1 spinal muscular ... gene therapy ...
Spinal muscular atrophy (SMA ... The disease results from a mutation to the ‘survival motor neurone’ (SMN) gene, causing a significant decrease in the expression of SMN protein.