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Genentech: 3-yr Data Reinforces Efficacy, Safety Of Evrysdi In Infants With Type 1 SMA

Genentech, a member of the Roche Group (RHHBY), reported new three-year data from the FIREFISH study, including one-year data from the open label extension. The company said study reinforced the long-term efficacy and safety of Evrysdi (risdiplam) in infants with symptomatic Type 1 spinal muscular atrophy. An estimated 91% of infants treated with Evrysdi were alive after three years of treatment. The Evrysdi-treated infants continued to improve or maintain motor functions.

"These long-term results in babies treated with Evrysdi are very encouraging, with the vast majority improving or maintaining motor functions after three years. Without treatment, they would typically not survive beyond two years of age," said Levi Garraway, Genentech's chief medical officer and head of Global Product Development.

The FIREFISH study evaluated Evrysdi in infants aged 1-7 months with Type 1 spinal muscular atrophy.

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