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Roche Reports Results From Evrysdi Study In Infants With Type 1 Spinal Muscular Atrophy

Roche reported results from Evrysdi study in infants with type 1 spinal muscular atrophy. The data showed that treatment with Evrysdi at 12 months helped 90% of these infants survive without permanent ventilation and 33% sit without support for at least 5 seconds, which is not normally seen in the natural course of the disease. The treatment with Evrysdi increased the levels of survival of motor neuron protein by a median 1.9-fold from baseline in the high-dose cohort at 12 months.

The FDA approved Evrysdi in 2020 for the treatment of spinal muscular atrophy in adults and children 2 months of age and older. Evrysdi was granted PRIME designation by the European Medicines Agency in 2018.

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