Hi--Just wanted to introduce myself. I joined PPMD in January as the Research and Advocacy Director after 11 years at MDA running their translational research and drug development programs. At PPMD I'll be further developing work with drug companies, reviewing and growing the main research program and participating in advocacy intiatives. I will also try to jump in as much as possible to answer questions about research and track down information about new treatments/therapies that anyone hears about. One of my first projects along this line is to get to the bottom of this stem cell report from Costa Rica. If anyone posts something that needs a response from me and you don't hear from me, you can always send me a nudge at email@example.com. I'm really excited about the chance to focus exclusively on Duchenne and am looking forward to hearing from all of you.
Thanks for all the warm welcomes--I'll try to keep everyone up to date to the best of my knowledge. As for the delays in getting FDA approval for exon-skipping in the U.S., I'm not sure what the current issue is, but initially there were problems complying with the FDA's request for tox data. There's a weird technical issue that was making it almost impossible for AVI to get the kind of tox data the FDA was requesting and they had basically hit an impasse. It's not clear to me if that particular technical issue has been resolved (the company brought on new management as many of you might know and seems to be in a much better place from that standpoint). But we reviewed this project several times when I was at MDA and chose not to fund it until the regulatory issues were resolved--we didn't want to tie up several million dollars in a project that wasn't moving. PPMD has held off for some of the same reasons. I know that's got to look frustrating from the outside if you don't know the back story, but it was mostly out of our hands. I'm trying to touch base with all of the companies working in the Duchenne space over the next few months and may have some updates I can report (still respecting the companys' need for confidentiality in some areas).
Hope that helps!