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Roche: new data in infants with SMA

( - Roche has presented new data from part 1 of the pivotal FIREFISH study of Evrysdi (risdiplam) in infants aged 2-7 months with type 1 symptomatic spinal muscular atrophy (SMA).

Results in infants treated with the therapeutic dose of Evrysdi (17/21) showed continued improvements and motor milestones.

Exploratory efficacy data showed that 88% of infants treated with Evrysdi were alive and did not require permanent ventilation at 2 years of age. 59% of infants were able to sit without support for at least 5 seconds.

In August, the FDA approved Evrysdi for the treatment of SMA in adults and children of at least 2 months of age.

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