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- W4237949110 abstract "Clinical trials in DMD require prolonged treatment durations to demonstrate slowing of the disease trajectory through functional tests. Quantitative MRS is a disease biomarker technology that may be an earlier predictor of drug effect; recent publications demonstrate its correlation to function. PhaseOut DMD is a Phase 2 open-label study evaluating ezutromid, a potential first-in-class utrophin modulator, administered to 40 ambulatory DMD patients who were all on stable corticosteroids. Primary endpoints (48 week) are MRS assessments. Two MRS endpoints were considered – water relaxation time (T2) and fat fraction (FF), for which drug effects have been reported at 3 and 12 months, respectively. Results of 24-week interim analysis have been reported. At baseline, mean (SD) T2 in the soleus muscle was 31.9 (1.9) ms (n=40); correlation coefficients (CC) with functional endpoints were all <0.3. Mean (SD) FF in the vastus lateralis (VL) muscle was 15.0% (13.38) (n=39). Baseline CC with functional endpoints (NSAA, 6MWD, 10 m walk/run and time to stand) ranged from -0.192 to 0.642. By week 12, a mean decrease from baseline in MRS-T2 in the soleus muscle was observed (-0.66 msec); by week 24, the decrease was statistically significant: average reduction was -0.86 ms (95% CI-1.44, -0.28; n=38). In untreated boys with DMD, MRS T2 does not change significantly over 1 year. An overall small increase in VL FF (+3.8%, n=37) was observed at 24 weeks; increases in FF are expected over time with annual average of +5 to +10%. The baseline data support the reported natural history regarding correlation of MRS FF to function. Interim data, after 24 weeks of ezutromid, indicate the potential for early identification of drug effects on disease progression. Results from the 48 week read-out will be presented." @default.
- W4237949110 created "2022-05-12" @default.
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- W4237949110 date "2018-10-01" @default.
- W4237949110 modified "2023-09-25" @default.
- W4237949110 title "DUCHENNE MUSCULAR DYSTROPHY – IMAGING AND BIOMARKERS" @default.
- W4237949110 doi "https://doi.org/10.1016/j.nmd.2018.06.064" @default.
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