Thanks for some of your insightful thoughts Wayne. Like you and some others have already mentioned, I agree that this is a great move on multiple fronts.
On the the clinical side, if our drug is save and efficacious then giving compassionate access to patients prior to approval should provide a meaningful benefit to the boys and continue to display positive results. It's even quite promising for our Phase2b results given that this was initiated by patients/investigators.
On the patient advocacy side this may help to improve our relationship with them and to show that we have a drug which may be efficacious. I think it's also a bonus that we can provide these free of charge. Shows that PER's got some class and compassion. Hopefully this bodes well for any future FDA/EMA approval.
Admittedly, all the news have been overshadowed by the sour taste of the CR, but I do think that our management proves that they've built a team who has got some good sense on the science and the clinical side of things. Let's see how this goes. Not long now.
Ann: Percheron launches post-trial access program, page-63
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