Started this discussion. Last reply by Colleen Jun 11, 2009. 6 Replies 0 Favorites
Started this discussion. Last reply by Christine Piacentino Apr 25, 2009. 8 Replies 0 Favorites
Posted on May 22, 2013 at 12:30pm 0 Comments 0 Favorites
Several weeks ago, I was invited to participate in a discussion focused on how large pharmaceutical companies should communicate with advocacy; how communication needs to be different in rare disease; and more specifically what advocates in rare diseases expect, want, and need from pharmaceutical companies. I was one of…
Posted on May 20, 2013 at 11:51am 0 Comments 1 Favorite
Wading through policy is no easy task. But imagine policy as the white lines on the road, the boundaries within which FDA operates. Building our case, showing how Duchenne fits into those white lines and demonstrating how and when Accelerated Approval makes perfect sense is the purpose of PPMD’s white paper. …
ContinuePosted on May 9, 2013 at 1:00pm 0 Comments 0 Favorites
Two weeks ago, PPMD proudly published Putting Patients First, a white paper outlining recommendations to speed responsible access to new therapies for Duchenne. In that short amount of time, the reaction to this white paper has been phenomenal. From interest by the media to accolades from industry and agencies, we have been truly humbled by the response we have received.
And while the…
ContinuePosted on May 2, 2013 at 4:16pm 0 Comments 0 Favorites
April 29-30 in Washington, D.C.
Recently I had the privilege of attending and participating in the Symposium on Best Practices in Clinical Study Design of Rare Diseases in Washington, D.C. The symposium was organized in collaboration with Children’s National Medical Center (CNMC), Clinical and Translational Science Institute of Children’s National (CTSI), The George Washington University, and Research in Pediatric Developmental Pharmacology Centers (RPDP). This…
ContinuePosted on May 1, 2013 at 10:30am 0 Comments 0 Favorites
CLINICAL TRIALS – The path to approval.
Typically we think about the Clinical Trial Process in the context of our kids. We hear about Phase I trials. Depending on the drug/biologic, many of these compounds will be required to be tested in healthy human subjects. Some Phase I trials have inclusion criteria, suggesting they are looking for healthy adults of a certain age to take a single or multiple doses of a compound or biologic. The purpose of the study…
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Hi Pat,
Do you know if there have been any studies on DMD boys and enzyme deficiencies? I met with a 5th generation doctor from India that says this is very common, and a cause of many problems with muscular dystrophy patients.
Thanks in advance...
Tammy
hi pat, just want to let u know that i am finding ppmd an absolutely brilliant resource and support. since my lovely wee nephew ben was diagnosed recently with duchenne, i feel as if i have become an expert on neuromuscular conditions. But we have to, as we have to take control for our boys. I think that you are an inspiration for all that you have achieved so far with pushing duchenne research abd treatment to where we are today. it is looking so much more promising. I recently sent u a friend request and would so appreciate to have u as a friend. God bless you pat and all your team. stephen beggs.
Hi Pat, I am happy for you to use photo of my boys. I also got an email fron Wil Nolan for the same request. Wishing you and your family a Merry Christmas...
Lisa Guest
I m Adnan Sarwar from Pakistan. i m suffering from muscular dystrophy
i m intrested to get some new research information and meet with other
md and their family for discuss issues related md.pls help me in this
connection.
Dear Pat,
Thank you so much for responding back. I would love to speak with you. What time tomorrow (Monday) would be a good time to talk to you? My phone # is 847-856-1201 and email address is momsangel0507@aol.com. I do have your cell phone # and will be happy to call you anytime you are available. Take care and thanks again.
Sincerely,
Julie Leston
Hi Pat,
I just wanted to let you know how happy I am to hear about the Super Bowl & the awareness that it will bring. I also wanted to let you know that my Minister Rev. Richard Young spoke of you & the article of you & PPMD in the New York Times. He said so many nice things about you & how your story touched his heart. His sermon was called "When God comes down". After the service I gave him a hug & thanked him for speaking about DMD & let him know that he has brought awareness to our congragation. Just wanted you to know that.
God Bless,
Suzanne Desmond
Orchard Park, NY (Buffalo, NY)
Hi Pat - Just read the article in The New Yorker. Excellent article. And whoever wrote it, sure did alot of background work. Really enjoyed it.
Happy birthday! I hope this year is the best year yet for you! I hope Tom is doing well too and of course, your girls. Thank you for your leadership, your tenacity and for all you do - Best -Char Burke
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