Results to Be Submitted to FDA with a Goal of Accelerating Review of Duchenne Therapies
PPMD has conducted a rigorous survey of more than 100 parents whose children have Duchenne, gathering invaluable perspectives as to treatment priorities and risk threshold based on realistic but hypothetical treatments - information that PPMD is encouraging regulators to use to accelerate review time and, ultimately, to speed access to…Continue
Added by PPMD on June 11, 2013 at 2:00pm — No Comments
Added by PPMD on June 7, 2013 at 8:30am — No Comments
Added by Kathi Kinnett on June 5, 2013 at 11:00am — No Comments
Added by Pat Furlong on May 30, 2013 at 1:26pm — No Comments
In February 2013, a bipartisan group of Senators and Representatives introduced the MD-CARE Act Amendments of 2013 (S. 315 and H.R. 594). This legislation seeks to update the landmark MD-CARE Act law…Continue
Added by Ryan Fischer on May 29, 2013 at 11:30am — No Comments
PPMD President Pat Furlong and Vice President of Research Sharon Hesterlee are attending MDA’s Scientific Conference this week in Washington, D.C. Most of what was presented are results that were previously announced. However, there were some interesting presentations, as well as, results worth repeating, that we wanted to share. Sharon and Pat will continue to blog from the conference:
From Pat Furlong
Interview with Thomas Voit
While the content…Continue
Below is the latest update PPMD has received from Dr. Rohit Batta, Global Medical Affairs and Patient Relations Leader in GSK's Rare Disease Unit :
The purpose of this communication is provide an update to you, as you have requested, on GSK’s plans for providing the option of continued access to drisapersen for boys completing the US Ph IIb 48 week study (DMD114876)…
Added by PPMD on April 24, 2013 at 9:30am — No Comments
Added by Pat Furlong on April 22, 2013 at 12:00pm — No Comments
Guest post by Gail D. McVicker, Grandmother of Jake (10) and Ryan (6) - Pennsylvania
Duchenne: An Intergenerational Issue
Five years ago, I did not know the world of Duchenne muscular dystrophy existed. Before my young grandsons were officially diagnosed in 2008, my daughter told me that some genetic…Continue
As you know, each month we feature someone in the community with Duchenne, that has done something really special to raise awareness and help us in our fight. This month, we are featuring 17-year-old Matt Scheivert, who doesn’t have Duchenne, but has already decided that he wants to continue to work in…Continue
Added by Will Nolan on April 17, 2013 at 2:22pm — No Comments
This spring brings news of progress to the Duchenne community as Sarepta engages in talks with the Food and Drug Association (FDA) to determine if they agency will review an application for accelerated approval for the exon 51 skipping drug eteplirsen and releases its 74 month data…Continue
One interesting thing about silence is listening to…Continue
Added by PPMD on April 4, 2013 at 11:30pm — No Comments
A Call to Action for all NH Constituents!
Seacoast, New Hampshire- 8th grader Rebecca Gowing wanted to make a difference in her community. After learning that her sister’s classmate and friend, Lucas Currier, had Duchenne Muscular Dystrophy she took action to help raise funds and spread awareness, creating Art for A Cure…Continue
Added by Ryan Fischer on April 2, 2013 at 9:00pm — No Comments
Hello Duchenne community!
Summer is just around the corner and with that comes sunshine, fireflies and PPMD’s Annual Connect Conference.
We are excited to be back in Baltimore, MD at the beautiful Marriott Waterfront this year and really excited about this year’s program.
Added by Kimberly Galberaith on April 2, 2013 at 11:30am — No Comments
Parliamentary Duchenne Rare Exon Meeting Report 26th March 2013
In Spring of this year, PPMD and other Duchenne stakeholders from around the world, convened a meeting with the UK's Shadow Minister of Health, Andrew Burnham, at Parliament. The purpose was focused on Antisense oligonucleotides (exon skipping) and developing an expedited pathway for all…Continue
Added by Pat Furlong on March 27, 2013 at 4:30pm — No Comments
Parent Project Muscular Dystrophy (PPMD) has awarded University of Minnesota researcher Dr. Rita Perlingeiro a grant for $220,000 to develop a therapeutic approach for Duchenne using a new type of "adult-derived" stem cell called "induced pluripotent stem cells" or "iPSCs." iPSCs can be created from non-controversial sources like skin, have the ability to multiply indefinitely, and can be coaxed to become many different kinds of tissue types.
Summit's Press Release:
Summit today outlines its future plans for the continued development of utrophin modulators for the treatment of DMD. Utrophin modulation is a disease modifying approach that has the potential to treat all genetic forms of DMD. Summit’s…
Added by PPMD on March 21, 2013 at 9:55am — No Comments
Added by Will Nolan on March 15, 2013 at 11:00am — No Comments
Help Bring Run for Our Sons to your own backyard.
I hear it all the time, “I want to do a fundraiser but not sure what to do or where to start” – I ask “have you thought of doing a 5K or fun run in your community?” – The rest is Run for Our Sons history!
5Ks and fun runs are fast becoming one of PPMD’s…Continue
Added by Ryan Fischer on March 14, 2013 at 1:30pm — No Comments