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 sharon@parentprojectmd.org.  I'm really excited about the chance to focus exclusively on Duchenne and am looking forward to hearing from all of you.

 

Best Wishes,

Sharon Hesterlee

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Hi Sharon,

Thank you for your intro. So glad you've jumped onboard since DMD is extremely complicated to the average mom. I look forward to reading your posts !!
cheryl
Congratulations on the new job - we're glad you're here!
Will you also be able to answer questions about pharma and FDA approvals? Some of us would like to know why the exon skipping trials are still on hold, pending FDA approval, here in the US... AVI/Columbus trial was planned for March and Prosensa/GSK's was planned for "early" 2010; it's February and no news unfortunately.

Good luck in the new job!
Glad to have you with us,We're in such need for people of noledge like you.Please give us updates on what's going on really,I personaly don't understand no more what really hapening,,,
Thank you,
Welcome - It is very exciting that you are on board on PPMD. I hope to meet you in Denver in June. Take care.
Sharon,

Thank you so much for taking the time to introduce yourself, Pat gave us an excellent aproach of who you are and we are happy to have you on our team.
Please keep us informed about three things, exon skipping (Avi and Prosensa) timings, Utrophin (Biomarin) and Project Catalyst.

Best vibes from Peru.
JP.
Hi sharon,
Glad to have you with us. Will be looking forward from you on updates on exon skipping, Utrophin and Stem cell part.

Wish you all the best.

Raktim
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!
Sharon
Hi
Welcome!
I would like to know if someone in US is looking at combination of (ibuprofen+ISDN (isosorbide dinitrate)) or Losartin + I believe some other drug to replace steroids. Thanks.
Thanks for the introductions Sharon. We are very glad that you decided to work for PPMD:) Do you have any information on the progress if any that Prosensa and GSK are making on getting their trials set up and what they need to do to bring them to the US?

Sharon Hesterlee said:
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!
Sharon
I can tell you that before I left MDA, MDA Venture Philanthropy (MVP) funded two drug studies to develop steroid replacements...both drugs targeted the inflammatory cascade without stimulating the hormone receptor, which is likely to be the cause of many side effects. Both companies, Catabasis and Validus, are small start-ups (Validus is Eric Hoffman's company) and they are still looking at efficacy in mouse models. I haven't heard anything about the ibuprofen combo you mention, but it doesn't mean that someone isn't trying it...at MDA I was focused on about 40 different neuromuscular diseases, so you guys might be more up to speed than I am on some of the more obscure strategies--at least for now!
I will let you know any updates on Prosensa and AVI as soon as I have them!

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