As next month’s US Food and Drug Administration (FDA) decision on Roche’s spinal muscular atrophy (SMA) drug looms large, the company has released new data on its efficacy. The results show that over 90% of children who start treatment at or before seven months of age survived. In addition, 32 out of 48 children maintained the ability to sit upright without external support.
According to Lei Lei Wu of Endpoints News, “Infants with SMA have a genetic mutation to their SMN1 gene, which encodes a protein crucial to muscle movement (SMN literally stands for “survival motor neuron”). Children with SMA may struggle with sitting up, swallowing, or breathing. Evrysdi works by promoting the other SMN gene, SMN2, to make more of that essential protein.”
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(Source: Endpoints News, April 29th, 2022)