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F. Hoffmann-La Roche Ltd: Roche receives positive CHMP opinion for Evrysdi, the first and only at home spinal muscular atrophy (SMA) treatment with proven efficacy in adults, children and infants two months and older


F. Hoffmann-La Roche Ltd: Roche receives positive CHMP opinion for Evrysdi, the first and only at home spinal muscular atrophy (SMA) treatment with proven efficacy in adults, children and infants two months and older
More than 2,500 patients now treated with Evrysdi in clinical trial, compassionate use and real-world settings
Evrysdi is approved in seven countries, submitted in 30 more
Basel, 26 February 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Evrysdi (risdiplam) 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. SMA is a leading genetic cause of death in infants and 5q SMA is the most common form of the disease. SMA causes progressive muscle weakness and atrophy, and significant unmet need rema ....

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