SRPT Duchenne Drug trial hits 5 year mark, page-37

The subject of this post was to make the point that there is another company that is facing the same predicament as Prana with a safe drug that patients want and feel that works for them. This is exactly what the orphan drug priority pathway is intended for. Put the finer points on efficacy after you have allowed access for the people that want it, feel that helps them and are facing dire circumstances. There is so much uncertainty in the cases of AD and HD about whether or not the current methods for assessing cognition are adequate (and they are not) that we should rely more on what the patients say and feel. There are so many drugs on the market right now that are way less safe than PBT2 or the SRPT drug.