Kristen Elaine

Thanks Michael! I am aware of all of the different drugs in development, as well as their associated clinical trials and the data findings therein. What I was curious about was, why is there the idea among the adult population that Zolgensma will ever be something an adult SMA affected individual can receive? While I mentioned that I am familiar…[Read more]

DeAnn- I was considering the same as you mentioned! I’m also not confident that the label of Zolgensma will be expanded to include adults, additionally considering the high potential for immunity to the viral vector among the adult population.  While the potential efficacy of primary gene replacement (SMN1 primary gene vs the secondary SMN2, non…[Read more]

So I’m curious, is there actually any reason to even suspect that Zolgensma will really be applicable to adults? Not older adolescents or essentially anyone still considered Pediatric, but to adults? The last I really read in to it, I remember seeing that there were trials being done to test the ability of zolgensma to be administered via…[Read more]