Roche touts 2-year ef­fi­ca­cy da­ta for SMA ther­a­py Evrys­di; NIH funds new net­work to study flu and virus­es with ‘pan­dem­ic po­ten­tial’

Two years af­ter ther­a­py, 61% of ba­bies with spinal mus­cu­lar at­ro­phy Type 1 treat­ed with Roche’s Evrys­di (ris­diplam) were able to sit up with­out sup­port for at least five sec­onds com­pared with just 29% of pa­tients af­ter a year, the drug­mak­er said Thurs­day.

As part of the FIRE­FISH-2 Phase III study, re­searchers test­ed Evrys­di’s abil­i­ty to help 41 in­fant pa­tients sit up with­out sup­port at 5 and 30 sec­onds. Da­ta showed that 44% of pa­tients were able to sit with­out sup­port for 30 sec­onds at the two-year check-in com­pared with 17% af­ter one year.

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