i feel like the preference to titrate to dose has allowed these US families to largely manage the GI issues that were so prevalent in the trials. with that in mind, I wonder if going to full dose straight away in the trials really masked the true efficacy of trofinetide by poorer QOL metrics in trial participants compared to current patients. i imagine if the trial families were battling acute diarrhoea and nausea, they may have missed some of the smaller nuanced improvements that current families are reporting all the time.
it will be really interesting to some of the 6/12 month QOL and physician feedback when it's released.
i feel like the preference to titrate to dose has allowed these...
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