Nearly all patients in this cohort achieved a clinically meaningful >3-point increase during the study period, demonstrating a consistent response to the gene therapy. from Front page feed https://ift.tt/2WWn6gj
Catabasis’s small molecule NF-kB inhibitor showed promise in phase 2, and the phase 3 PolarisDMD study (NCT03703882) of edasalonexant is fully enrolled and expected to read out in late 2020. from Front page feed https://ift.tt/3anrvMV
Although the drug requires no CYP2C9 genotyping or first-dose observation, an up-titration scheme should be used due to the risk of transient cardiac conduction issues. from Front page feed https://ift.tt/3amkLPl