Clinical Data
The new MRS data showed a statistically significant decrease from baseline in the T2-relaxation time in the soleus (calf muscle) in patients (n=38) treated with ezutromid. The mean decrease was -0.861 milliseconds from baseline to 24 weeks (31.850 milliseconds to 30.989 milliseconds, 95% CI, -1.440, -0.281). The soleus is one of the most reliable leg muscles for monitoring disease progression via T2 relaxation time in DMD with increases shown to correlate to loss of functional ability.[1,2] Published natural history data show that T2 relaxation times increase with disease progression due to the relentless cycle of muscle damage and repair leading to inflammation.[1,2] A mean decrease of -0.470 milliseconds in MRS-T2 was also observed in the vastus lateralis (thigh muscle) in ezutromid-treated patients (n=37) from baseline to 24 weeks (32.265 milliseconds to 31.795 milliseconds, 95% CI, -1.158, 0.218).