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Risdiplam for treating spinal muscular atrophy

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2 Information about risdiplam

Marketing authorisation indication

2.1 Risdiplam (Evrysdi, Roche) is indicated for 'the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies'.

Dosage in the marketing authorisation

2.2 The dosage schedule is available in the summary of product characteristics.

Price

2.3 The list price is £7,900 per 60mg/80ml vial. The company has a commercial arrangement, which would have applied if the technology had been recommended.