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Treatment with the Catabasis Pharmaceuticals small molecule oral NF-kB inhibitor resulted in improvements in the 10-meter walk/run scores, time-to-stand scores, and 4-stair climb scores in 31 steroid-naïve boys with DMD aged 4 to 7 years.

The professor of chemistry and chemical biology at Harvard University and Blavatnik National Laureate discussed the work she and colleagues have done on microbiome metabolism and what upcoming plans to pursue this further could achieve.