Strong Evrysdi data keep coming in SMA

15 April 2021

PTC Therapeutics (Nasdaq: PTCT) has announced new data showing continued improvements in motor milestones in infants with type 1 spinal muscular atrophy (SMA) after two years of treatment with Evrysdi (risdiplam).

Results from Part 2 of the pivotal FIREFISH trial demonstrated that infants treated with Evrysdi obtained increases in survival and sustained improvements in milestones including head control, sitting, rolling over, and further developing towards acquiring the ability to stand, and walk.

At 24 months, results showed increased survival rates, with 93% of infants alive and 83% not on permanent ventilation. An improved and maintained ability to feed orally was seen in 92% of patients, with 44% showing improved motor function, including the ability to sit without support. Reduced hospitalization was also seen, with 34% not requiring admission.

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