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Earlier this month the Medicines and Healthcare products regulatory agency approved a license extension for Risdiplam (Roche) to include people of all ages. Prior to this extension, the recommendation to prescribe Risdiplam was indicated only for people ages 2 months and over.


“We are really glad to hear that children aged 2 months and under will now be able to access Risdiplam as we know early treatment intervention can provide significant positive impacts to babies” says Portia Thorman, Advocacy Lead at SMA UK.


Risdiplam is recommended as an option for treating 5q spinal muscular atrophy (SMA) In people of all ages with a clinical diagnosis of SMA types 1, 2 or 3 or with pre-symptomatic SMA and 1 to 4 SMN2 copies.

Read more on the updated guidance through the NICE website here.