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Genentech, a Roche company, announced new positive data for the oral therapy Evrysdi (risdiplam) in patients with spinal muscular atrophy (SMA)
SMA is a serious, progressive, and potentially fatal neuromuscular disease
Evrysdi is a small molecule mRNA splicing regulator targeting the SMN2 gene, jointly developed by Genentech and PTC Therapeutics
In a Phase 2/3 clinical trial called SUNFISH, in patients treated with Evrysdi, the improvements observed in total Motor Function Scale-32 (MFM-32) scores after the first year of treatment were still seen at the third year maintain
In addition, for the first time, the researchers conducted an external comparative analysis of two years of data from the trial with a group of untreated patients
The latest interim results from another Phase 2 clinical trial in infants with SMA under two months who had not yet developed symptoms showed that most infants treated with Evrysdi for at least 12 months were able to stand and walk within the time frame typical of healthy infants
References:
[1] New Data for Genentech's Evrysdi (risdiplam) Demonstrate Long-Term Efficacy and Safety in a Broad Population of People With Spinal Muscular Atrophy (SMA).
